[House Hearing, 114 Congress]
[From the U.S. Government Publishing Office]


                       THE SCIENCE AND ETHICS OF
                    GENETICALLY ENGINEERED HUMAN DNA

=======================================================================

                                HEARING

                               BEFORE THE

                 SUBCOMMITTEE ON RESEARCH & TECHNOLOGY

              COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY
                        HOUSE OF REPRESENTATIVES

                    ONE HUNDRED FOURTEENTH CONGRESS

                             FIRST SESSION

                               __________

                             JUNE 16, 2015

                               __________

                           Serial No. 114-24

                               __________

 Printed for the use of the Committee on Science, Space, and Technology
 
 
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              COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY

                   HON. LAMAR S. SMITH, Texas, Chair
FRANK D. LUCAS, Oklahoma             EDDIE BERNICE JOHNSON, Texas
F. JAMES SENSENBRENNER, JR.,         ZOE LOFGREN, California
    Wisconsin                        DANIEL LIPINSKI, Illinois
DANA ROHRABACHER, California         DONNA F. EDWARDS, Maryland
RANDY NEUGEBAUER, Texas              SUZANNE BONAMICI, Oregon
MICHAEL T. McCAUL, Texas             ERIC SWALWELL, California
MO BROOKS, Alabama                   ALAN GRAYSON, Florida
RANDY HULTGREN, Illinois             AMI BERA, California
BILL POSEY, Florida                  ELIZABETH H. ESTY, Connecticut
THOMAS MASSIE, Kentucky              MARC A. VEASEY, TEXAS
JIM BRIDENSTINE, Oklahoma            KATHERINE M. CLARK, Massachusetts
RANDY K. WEBER, Texas                DON S. BEYER, JR., Virginia
BILL JOHNSON, Ohio                   ED PERLMUTTER, Colorado
JOHN R. MOOLENAAR, Michigan          PAUL TONKO, New York
STEVE KNIGHT, California             MARK TAKANO, California
BRIAN BABIN, Texas                   BILL FOSTER, Illinois
BRUCE WESTERMAN, Arkansas
BARBARA COMSTOCK, Virginia
DAN NEWHOUSE, Washington
GARY PALMER, Alabama
BARRY LOUDERMILK, Georgia
RALPH LEE ABRAHAM, Louisiana
                                 ------                                

                Subcommittee on Research and Technology

                 HON. BARBARA COMSTOCK, Virginia, Chair
FRANK D. LUCAS, Oklahoma             DANIEL LIPINSKI, Illinois
MICHAEL T. MCCAUL, Texas             ELIZABETH H. ESTY, Connecticut
RANDY HULTGREN, Illinois             KATHERINE M. CLARK, Massachusetts
JOHN R. MOOLENAAR, Michigan          PAUL TONKO, New York
BRUCE WESTERMAN, Arkansas            SUZANNE BONAMICI, Oregon
DAN NEWHOUSE, Washington             ERIC SWALWELL, California
GARY PALMER, Alabama                 EDDIE BERNICE JOHNSON, Texas
RALPH LEE ABRAHAM, Louisiana
LAMAR S. SMITH, Texas
                           
                           C O N T E N T S

                             June 16, 2015

                                                                   Page
Witness List.....................................................     2

Hearing Charter..................................................     3

                           Opening Statements

Statement by Representative Barbara Comstock, Chairwoman, 
  Subcommittee on Research, Committee on Science, Space, and 
  Technology, U.S. House of Representatives......................     5
    Written Statement............................................     5

Statement by Representative Daniel Lipinski, Ranking Minority 
  Member, Subcommittee on Research, Committee on Science, Space, 
  and Technology, U.S. House of Representatives..................     6
    Written Statement............................................     7

Statement by Representative Lamar S. Smith, Chairman, Committee 
  on Science, Space, and Technology, U.S. House of 
  Representatives................................................     8
    Written Statement............................................     9

                               Witnesses:

Dr. Victor J. Dzau, President, Institute of Medicine, the 
  National Academy of Sciences
    Oral Statement...............................................    10
    Written Statement............................................    13

Dr. Jennifer Doudna, Professor of Biochemistry and Molecular 
  Biology, University of California,
    Oral Statement...............................................    18
    Written Statement............................................    20

Dr. Elizabeth McNally, Professor of Genetic Medicine, Professor 
  in Medicine-Cardiology and Biochemistry and Molecular Genetics; 
  Director, Center for Genetic Medicine, Northwestern University
    Oral Statement...............................................    23
    Written Statement............................................    25

Dr. Jeffrey Kahn, Professor of Bioethics and Public Policy; 
  Deputy Director for Policy and Administration, Berman Institute 
  of Bioethics, Johns Hopkins University
    Oral Statement...............................................    32
    Written Statement............................................    34

Discussion.......................................................    40

             Appendix I: Answers to Post-Hearing Questions

Dr. Elizabeth McNally, Professor of Genetic Medicine, Professor 
  in Medicine-Cardiology and Biochemistry and Molecular Genetics; 
  Director, Center for Genetic Medicine, Northwestern University.    64

            Appendix II: Additional Material for the Record

Statement by Representative Eddie Bernice Johnson, Ranking 
  Member, Committee on Science, Space, and Technology, U.S. House 
  of Representatives.............................................    66

 
                       THE SCIENCE AND ETHICS OF
                    GENETICALLY ENGINEERED HUMAN DNA

                              ----------                              


                         TUESDAY, JUNE 16, 2015

                  House of Representatives,
                    Subcommittee on Research and Technology
               Committee on Science, Space, and Technology,
                                                   Washington, D.C.

    The Subcommittee met, pursuant to call, at 2:16 p.m., in 
Room 2318 of the Rayburn House Office Building, Hon. Barbara 
Comstock [Chairwoman of the Subcommittee] presiding.
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT] 

    Chairwoman Comstock. The Subcommittee on Research and 
Technology will come to order. Without objection, the Chair is 
authorized to declare recesses of the Subcommittee at any time.
    And without objection, the gentleman from California, Mr. 
Sherman, is authorized to participate in today's hearing.
    Good afternoon, and welcome to this hearing entitled ``The 
Science and Ethics of Genetically Engineered Human DNA.''
    And I believe we also would like to welcome Representative 
Abraham to his first Science Committee hearing. Dr. Abraham, we 
are happy to have you join the Research and Technology 
Subcommittee and we look forward to having the benefit of your 
expertise.
    Now, in front of you are packets containing the written 
testimonies, biographies, and truth-in-testimony disclosures 
for today's witnesses.
    I now recognize myself for an opening statement.
    Biotechnology--the engineering of genetic material in 
living beings and plants--has transformed modern medicine and 
agriculture. Rapid advances in biotech research have brought 
great opportunities for new medical treatments and products, 
and simultaneously have also raised questions about possible 
ethical implications and safety issues.
    Today, we are here to discuss the science and ethics of the 
most recent and eye-opening development in biotechnology: human 
genome-editing. This research has been a major topic of news 
and editorials in recent months. New tools that allow a gene to 
be deleted, inserted, or replaced by a different piece of DNA 
are becoming more cost-effective and simpler to execute.
    In April it was reported that for the first time a team of 
Chinese scientists had attempted to edit the genome of human 
embryos. The report raised concerns for many scientists and 
policymakers about the safety and ethics of using these new 
technologies on human DNA. Many prominent scientists have 
called for a better framework to be developed for responsible 
use of the technology.
    I look forward to learning more from our witnesses today 
who will provide an overview of the science behind these new 
technologies, help us examine the implications and risks, and 
explore what the next steps should be for building the right 
kind of framework for utilizing the technology. They will also 
help us answer how the United States can be a leader and 
provide scientific and ethical leadership in this arena.
    [The prepared statement of Chairwoman Comstock follows:]

                   Prepared Statement of Subcommittee
                      Chairwoman Barbara Comstock

    Biotechnology--the engineering of genetic material in living beings 
and plants--has transformed modern medicine and agriculture.
    Rapid advances in biotech research have brought great opportunities 
for new medical treatments and products, and simultaneously have also 
raised questions about possible ethical implications and safety issues.
    Today, we are here to discuss the science and ethics of the most 
recent and eye-opening development in biotechnology: human genome-
editing.
    This research has been a major topic of news and editorials in 
recent months. New tools that allow a gene to be deleted, inserted, or 
replaced by a different piece of DNA are becoming more cost-effective 
and simpler to execute.
    In April, it was reported that for the first time a team of Chinese 
scientists had attempted to edit the genome of human embryos. The 
report raised concerns for many scientists and policy makers about the 
safety and ethics of using these new technologies on human DNA.
    Many prominent scientists have called for a better framework to be 
developed for responsible use of the technology.
    I look forward to learning more from our witnesses today who will 
provide an overview of the science behind these new technologies, help 
us examine the ethical implications and risks, and explore what the 
next steps should be for building a responsible framework for utilizing 
the technology. They will also help us answer how the United States can 
provide scientific and ethical leadership in this arena.

    Chairwoman Comstock. So I now recognize the Ranking Member, 
the gentleman from Illinois, for his opening statement.
    Mr. Lipinski. Thank you, Chairwoman Comstock, for holding 
this hearing on the science and ethics of new gene editing 
technologies.
    I want to thank all the witnesses for being here today and 
look forward to your testimony.
    Although we're talking about gene editing technologies that 
are very new, it's important to mention that humans have been 
altering the genomes of species through selective breeding for 
thousands of years. And since the 1970s, it has been possible 
to directly manipulate DNA, which led to a biotechnology 
revolution and significant economic growth.
    Then we had the Human Genome Project to sequence the human 
genome, and it was coordinated by the Department of Energy and 
the National Institutes of Health. The full human genome was 
sequenced in 2003, opening up whole new possibilities for 
diagnosing and treating diseases. One such pathway led to the 
invention of the CRISPR technology.
    Thanks to new gene editing technologies, which include 
CRISPR, we're able to add, remove, and replace DNA bases. They 
can be thought of as search-and-replace tools for DNA. They're 
incredibly powerful technologies that have the potential to 
transform the healthcare, energy, and agricultural sectors. 
Although new, these technologies were the outgrowth of decades 
of fundamental research, some of which was supported by the 
National Science Foundation.
    We are here today because a Chinese research group recently 
published a paper in which they used these technologies to try 
to modify human embryos. That paper highlights scientific and 
ethical issues with these technologies, especially if they are 
being used to modify human germline cells as opposed to adult 
somatic cells.
    I look forward to hearing about the science behind these 
technologies, as well as how the United States can be a leader 
in addressing the safety and ethical concerns associated with 
them.
    I understand the National Academies has launched a major 
initiative around human gene editing technologies. In the 
1970s, the National Academies played a similar role dealing 
with the then-new biotechnologies, and I look forward to 
hearing more about what they're planning to do concerning these 
new gene editing technologies. I also look forward to hearing 
about some of the potential nonhuman applications.
    [The prepared statement of Mr. Lipinski follows:]

                   Prepared Statement of Subcommittee
                Minority Ranking Member Daniel Lipinski

    Thank you Chairwoman Comstock for holding this hearing on the 
science and ethics of new gene editing technologies. I want to thank 
all the witnesses for being here this afternoon and I look forward to 
hearing your testimony.
    Although we are talking about gene editing technologies that are 
very new, it is important to mention that humans have been altering the 
genomes of species through selective breeding for thousands of years. 
Since the 1970s, it has been possible to directly manipulate DNA, which 
led to a biotechnology revolution and significant economic growth. Then 
we had the Human Genome Project to sequence the human genome that was 
coordinated by the Department of Energy and the National Institutes of 
Health. The full human genome was sequenced in 2003, opening up whole 
new possibilities for diagnosing and treating diseases. One such 
pathway led to the invention of the CRISPR technology.
    Thanks to new gene editing technologies, which include CRISPR, we 
are able to add, remove, and replace DNA bases. They can be thought of 
as ``search and replace'' tools for DNA. They are incredibly powerful 
technologies that have the potential to transform the health care, 
energy, and agricultural sectors. Although new, these technologies were 
the outgrowth of decades of fundamental research, some of which was 
supported by the National Science Foundation. We are here today because 
a Chinese research group recently published a paper in which they used 
these technologies to try to modify human embryos. That paper 
highlights scientific and ethical issues with these technologies, 
especially if they are being used to modify human germline cells as 
opposed to adult somatic cells.
    I look forward to hearing about the science behind these 
technologies as well as how the United States can be a leader in 
addressing the safety and ethical concerns associated with them. I 
understand that the National Academies has launched a major initiative 
around human gene editing technologies. In the 1970s, the National 
Academies played a similar role dealing with the then-new 
biotechnologies and I look forward to hearing more about what they are 
planning to do concerning these new gene editing technologies. I also 
look forward to hearing about some of the potential non-human 
applications.
    Now I would like to yield my remaining time to my colleague from 
Illinois, Mr. Foster, who is very interested in this topic and helped 
organize today's hearing.

    Mr. Lipinski. With that, I'd like to yield my remaining 
time to my colleague and neighbor from Illinois, Dr. Foster, 
who was very interested in this topic and helped to organize 
today's hearing.
    Mr. Foster. Thank you, and I'd like to thank the whole 
Research and Technology Subcommittee, including the Chair, 
Congresswoman Comstock, and Ranking Member Lipinski for 
allowing me to join you here today. And similarly, I wanted to 
thank Chairman Smith and Ranking Member Johnson for agreeing to 
hold this hearing. And a very special thank you to the 
witnesses for taking their time out for this very important 
issue.
    It is rare that prominent members of the scientific 
community come together to warn our leaders of technological 
breakthroughs that our legal system and society may not be 
prepared for, and yet, this is exactly what appears to be 
happening with recent discoveries in genetic editing tools.
    As the last Ph.D. scientist in Congress, I am afraid I've 
served a sort of a lightning rod for many of these warnings and 
I take them very seriously.
    I want to commend the National Academy of Sciences and the 
Institute of Medicine for the launch of their major initiative 
on human gene editing, and I want to make sure that Congress 
does everything constructive that it can to make sure that this 
is handled responsibly.
    There is the possibility of very great benefits from these 
new technologies, and what makes them really revolutionary is 
what they can mean for humans, for example, replacing bone 
marrow of someone suffering from sickle cell disease with a 
modified version of their own marrow with the genetic defect 
removed.
    However, if genetic modifications are made to so-called 
germline cells--these are sperm, eggs, embryos--then the 
modifications will be carried forward to future generations, 
which has implications that we need to carefully consider. 
We're on the verge of a technological breakthrough that could 
change the future of mankind and we must not blindly charge 
ahead.
    Thank you, and I yield back my time.
    Mr. Lipinski. I will just conclude. I agree with Dr. Foster 
and it's great to see that we have so many people here at this 
hearing. And it's a very important issue that we really need to 
consider deeply, so I thank the Chairwoman and the Chairman of 
the Full Committee, Chairman Smith, for holding this hearing 
today, and I'll yield back.
    Chairwoman Comstock. Thank you.
    And I now recognize the Chairman of the Full Committee, Mr. 
Smith.
    Chairman Smith. Thank you, Madam Chair.
    I do look forward, as do the others, to today's discussion 
on a new development in biology, which has been called ``a game 
changer,'' ``revolutionary,'' ``powerful,'' and ``a major issue 
for all humanity.''
    The new discoveries in genetically engineering human DNA 
offer potential cures for devastating genetic disorders. But 
the speed at which these new, simpler, and cheaper technologies 
are being used in the lab also presents ethical and health 
concerns. Most of the scientific community members have been 
clear: the science and ethics of this new technology must be 
resolved in order to prevent dangerous abuses and unintended 
consequences.
    A recent report from China, where teams of researchers have 
begun to experiment with engineering DNA in human embryos, is 
alarming. This is an area where the United States can and 
should provide scientific and moral leadership. We need to 
better understand the technology and procedures being used so 
that we can ensure patients are treated in the safest and most 
ethical manner possible.
    An April editorial in Science magazine called for a prudent 
path forward for genomic engineering. It recommended a 
moratorium on further research, while creating public forums 
for scientists, ethicists, and policymakers to discover the--to 
discuss ``the attendant ethical, social, and legal implications 
of genome modification.'' This is why it is important that the 
House Science Committee is holding the first Congressional 
hearing on this profound and complex subject.
    The purpose of the Science Committee is to explore the 
significance of scientific discoveries, as well as their 
potential implications for humankind. But we also must always 
be conscious of the potential ethical and moral issues raised 
by previously unimagined scientific breakthroughs. We must take 
the lead in reviewing new and innovative areas of science, such 
as genetically engineered DNA.
    So I look forward, Madam Chair, to this informative 
discussion today, and we have an excellent panel of witnesses 
to hear from as well.
    And I'll yield back.
    [The prepared statement of Chairman Smith follows:]

   Prepared Statement of Committee on Science, Space, and Technology
                          Chairman Lamar Smith

    Thank you Madam Chair. I look forward to today's discussion on a 
new development in biology, which has been called ``a game changer,'' 
``revolutionary,'' ``powerful,'' and ``a major issue for all 
humanity.''
    The new discoveries in genetically engineering human DNA offer 
potential cures for devastating genetic disorders. But the speed at 
which these new, simpler and cheaper technologies are being used in the 
lab also presents ethical and health concerns.
    Most of the scientific community members have been clear: the 
science and ethics of this new technology must be resolved in order to 
prevent dangerous abuses and unintended consequences.
    A recent report from China, where teams of researchers have begun 
to experiment with engineering DNA in human embryos, is alarming. This 
is an area where the United States can and should provide scientific 
and moral leadership.
    We need to better understand the technology and procedures being 
used so that we can ensure patients are treated in the safest and most 
ethical manner possible.
    An April editorial in Science Magazine called for a prudent path 
forward for genomic engineering. It recommended a moratorium on further 
research, while creating public forums for scientists, ethicists and 
policy makers to discuss ``the attendant ethical, social, and legal 
implications of genome modification.''
    This is why it is important that the House Science Committee is 
holding the first congressional hearing on this profound and complex 
subject.
    The purpose of the Science Committee is to explore the significance 
of scientific discoveries as well as their potential implications for 
humankind.
    But we also must always be conscious of the potential ethical and 
moral issues raised by previously unimagined scientific breakthroughs.
    We must take the lead in reviewing new and innovative areas of 
science, such as genetically engineered DNA.
    I look forward to an informative discussion with our distinguished 
panel of witnesses.

    Chairwoman Comstock. Thank you.
    And if there are Members who wish to submit additional 
opening statements, your statements will be added to the record 
at this point.
    Now, at this time I would like to introduce our witnesses. 
Dr. Victor Dzau is the President of the National Academies 
Institute of Medicine and the James B. Duke Professor of 
Medicine at Duke University. Dr. Dzau has received many honors, 
including the Distinguished Scientist Award from the American 
Heart Association. He earned his undergraduate and medical 
degrees from McGill University and holds eight honorary 
doctorates.
    Our second witness today is Dr. Jennifer Doudna. I think I 
got that. Dr. Doudna is Professor of Molecular and Cell Biology 
and Professor of Chemistry at U.C. Berkeley. A member of the 
National Academy of Sciences, Dr. Doudna is the recipient of 
several awards, including the NSF Waterman Award and the 2015 
Breakthrough Prize for Life Sciences. Dr. Doudna earned her 
undergraduate degree in biochemistry from Pomona College and 
her Ph.D. in biological chemistry from Harvard University.
    I now recognize the gentleman from Illinois, Mr. Lipinski, 
to introduce our next witness.
    Mr. Lipinski. Thank you.
    As a Northwestern University alumnus, I'm very excited to 
have Dr. McNally here today. To say an aside, Dr. Dzau, I'm 
also an alum of Duke University, and unfortunately for Dr. 
Doudna, an alum of Stanford also.
    Dr. McNally is the Director of the Center for Genetic 
Medicine and Professor in the Departments of Medicine and 
Biochemistry at Northwestern University's Feinberg School of 
Medicine. She is a cardiologist who specializes in inherited 
forms of heart disease. Dr. McNally's research has identified 
genes and mechanisms for how genetic lead to heart and muscle 
disease. She has an undergraduate degree in biology and 
philosophy from Barnard College at Columbia University and an 
M.D. and Ph.D. from the Albert Einstein College of Medicine.
    It is my pleasure to welcome Dr. McNally to our committee 
and look forward to her testimony.
    Chairwoman Comstock. Okay. And our final witness is Dr. 
Jeffrey Kahn, the Robert Henry Levi and Ryda Hecht Levi 
Professor of Bioethics and Public Policy at the Johns Hopkins 
Berman Institute of Bioethics and a Professor in the Department 
of Health Policy and Management at the Johns Hopkins Bloomberg 
School of Public Health. Dr. Khan received his bachelor's in 
microbiology from the University of California, Los Angeles, 
his master's in public health from Johns Hopkins, and his Ph.D. 
in philosophy and bioethics from Georgetown University.
    In order to allow time for discussion, we would ask that 
you limit your testimony to five minutes and your entire 
written statement will be made part of the record.
    I now recognize Dr. Dzau for five minutes to present his 
testimony.

          TESTIMONY OF DR. VICTOR J. DZAU, PRESIDENT,

                     INSTITUTE OF MEDICINE,

                THE NATIONAL ACADEMY OF SCIENCES

    Dr. Dzau. Good afternoon, Chairman Smith, Chairwoman 
Comstock, Ranking Member Lipinski, and Subcommittee Members. As 
you heard, I'm Victor Dzau. I'm the President of the Institute 
of Medicine, which will soon be named the National Academy of 
Medicine on July 1.
    I'm pleased to be here on behalf of the National Academies 
of Sciences, Engineering, and Medicine. The Academies operate 
under a Congressional Charter signed by Abraham Lincoln to 
provide advice to the Nation on matters where science, 
technology, and medicine can solve complex challenges and 
thereby improve people's lives.
    Thank you for the opportunity to speak with you today about 
this very important matter of human gene editing and the major 
initiative that we have at the National Academies launched to 
help guide decision-making in this area.
    The Academies have an established record of leadership on 
advising on emerging and often controversial areas of science, 
particularly genetic research, such as recombinant DNA and stem 
cell research. Our initiative is marshaling the best scientific 
evidence, medical, ethical, legal, and other expertise to help 
you and the Nation obtain a thorough understanding of gene 
editing and its potential risks and benefits. Our work is 
intended to provide a solid foundation to help inform decisions 
and policies on this research.
    As you will hear from other witnesses today, gene editing 
technology holds great promise. In fact, powerful new tools 
such as CRISPR/Cas9 developed by my colleague Dr. Doudna and 
others, as well as other genetic engineering technology, allow 
researchers now to precisely modify the genetic makeup of any 
living organism, including humans. The possible benefit 
application for such technologies are many. They could offer a 
cure to devastating genetic diseases such as Huntington's 
disease, as you heard, or sickle cell anemia. It can help 
improve and understand the treatment of many other illnesses.
    These technologies also present complex challenges both to 
scientific and medical communities and to society as a whole. 
Of particular concern is the potential to make permanent 
modification to human DNA in nuclei of eggs, sperm, or human 
embryos that are then passed down to succeeding generations 
known as germline gene editing. Research that attempts to alter 
the human germline raises important issues in so many different 
ways about safety, risk, social, economic, ethical, and 
regulatory considerations. So although more remains to be done 
before these technologies can be deployed safely, their 
availability certainly intensifies this debate among scientists 
and physicians about such research.
    Here in the United States there are legislative 
prohibitions on the use of federal funds for research of human 
embryos and there exist constraints on such research when 
subject to oversight by the U.S. Food and Drug Administration 
or other government agencies. These constraints, however, do 
not apply to work done internationally without federal funds 
and without the intent to seek federal approval of any product 
of that research. So clearly, we have reached a critical 
juncture in genetic editing research and guidance is needed.
    The National Academies of Sciences, Engineering, and 
Medicine are prepared to provide that guidance based on an in-
depth review of science underlying gene editing, the potential 
benefits, and the valid concerns raised by this research. 
Toward that end, our initiative on human gene editing research 
is already underway. Just last week, we held the first meeting 
of a multidisciplinary advisory group that will help us steer 
our initiative and ensure the Academies' efforts are 
comprehensive, inclusive, and transparent.
    Since much of this research is done internationally, the 
Academies will convene a global summit to obtain multinational 
perspectives on recent scientific development in human gene 
editing and the associated ethical and governance issues. 
Concurrently, we'll appoint an expert committee to conduct a 
comprehensive study of the scientific underpinning and 
clinical, ethical, legal, and social implications of human gene 
editing. We hope that we will come up with recommendations 
which can inform the Nation for decisions in this area.
    All of us--scientists, physicians, policymakers, and 
public--want to do everything possible to ensure the scientific 
and medical breakthroughs benefit all of mankind and do no 
harm. The Academies are certainly ready to help.
    I would be very happy to answer any questions the 
Subcommittee may have. Thank you.
    [The prepared statement of Dr. Dzau follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
            
    Chairwoman Comstock. And Dr. Doudna.

               TESTIMONY OF DR. JENNIFER DOUDNA,

        PROFESSOR OF BIOCHEMISTRY AND MOLECULAR BIOLOGY,

               UNIVERSITY OF CALIFORNIA, BERKELEY

    Dr. Doudna. Good afternoon, Chairwoman Comstock and the 
rest of the Members of the Committee. It's a great pleasure for 
me to be here and have the opportunity to talk with you about 
science that I've been involved with from its origin and 
involved in leading the discussion of where it's going.
    I wanted to start by saying that this is research that 
originated as a basic science project funded in part by the 
National Science Foundation. We did not aim to develop a genome 
editing technology but in the course of the experiments that we 
were doing, it became clear that what started as a study of a 
bacterial immune system, the way bacteria fight the flu, could 
actually be reengineered and re-harnessed really as a 
technology for changing sequences in the genomes of cells and 
whole organisms.
    I wanted to tell you a little bit more about the science 
behind this to explain a little bit about how it works and why 
it's revolutionary. So I think what really makes this distinct 
from other ways of manipulating DNA and cells is that it's a 
very simple system. It relies I would really make the analogy 
to software that you use for your computer. Here we have a 
protein called Cas9 that can be easily reprogrammed by using a 
short piece of nucleic acid called RNA that enables this 
protein to be directed to essentially any DNA sequence in a 
genome of an organism. And because genome sequencing has become 
very prevalent and is becoming less and less expensive, we have 
an exciting convergence of technologies that give us 
information about the entire genome in a cell or an organism 
and now a tool that allows scientists to change that sequence 
in a very precise fashion so we can do things, as was mentioned 
in the opening statements, like correct mutations that would 
otherwise lead to genetic disease.
    So this is, I think, a very exciting moment in biology. 
It's opened up a lot of opportunities for research, for 
clinical applications in the future, but it also raises various 
questions about the way that this technology should be employed 
going forward, and in particular in our discussion today the 
question of whether and when this technology should be employed 
to change the sequence in the human germline in eggs or sperm 
or embryos that would lead to a genetically modified person 
that would be a mutation that could be passed down to their 
children.
    And I realized fairly early on in our research that this 
technology was likely to be applicable in the human germline, 
and that led me to initiate a discussion initially with some--a 
fairly small group of scientists in California. We met in 
January of this year in the Napa Valley to discuss this very 
issue and we spent a day. We had--that small meeting included 
scientists, clinicians, as well as bioethicists to discuss the 
various issues around human germline editing, and that meeting 
resulted in a perspective that was published in Science 
magazine about two months ago that was referred to in the 
opening statements for--that called for a prudent path forward 
in any kind of clinical application of germline editing in 
humans.
    I do want to point out that our perspective favors research 
in this area. I think we feel as scientists that it's very 
important to have data so that we can make informed decisions 
about future potential applications, and I think this is a--I 
think many of us appreciate this is a technology that could be 
very helpful for people that have inherited genetic disorders. 
However, to ensure that any kind of application clinically in 
the human germline was safe and really was used in an ethical 
fashion, we do need to understand how this technology operates 
in those types of cells.
    Thank you.
    [The prepared statement of Dr. Doudna follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Chairwoman Comstock. Thank you.
    Now, we'll hear from Dr. McNally.

              TESTIMONY OF DR. ELIZABETH MCNALLY,

                 PROFESSOR OF GENETIC MEDICINE,

       PROFESSOR IN MEDICINE-CARDIOLOGY AND BIOCHEMISTRY

                    AND MOLECULAR GENETICS;

             DIRECTOR, CENTER FOR GENETIC MEDICINE,

                    NORTHWESTERN UNIVERSITY

    Dr. McNally. Thank you.
    On behalf of Northwestern University, I'd like to thank 
Chairwoman Comstock and Ranking Member Lipinski for inviting me 
here today.
    I'm Elizabeth McNally. I'm the Ward Professor of Genetic 
Medicine, and I direct the Center for Genetic Medicine at 
Northwestern. I'm a cardiologist and I specialize in providing 
care for patients and families with inherited forms of heart 
disease. Over the last decade, we've seen a dramatic increase 
in available genetic testing and we now routinely provide 
genetic diagnosis, risk assessment, and importantly, risk 
reduction for genetic diseases that affect the heart.
    Diseases like cystic fibrosis, Duchenne muscular dystrophy, 
sickle cell anemia are those that are caused by mutations in 
single genes. The gene editing techniques that we are here 
discussing offer the opportunity for permanent lifelong 
treatment of those disorders. With advances in DNA sequencing 
technology, it is now possible to sequence an individual genome 
in a day. For less than the cost of an MRI, a genome can be 
analyzed with high accuracy pinpointing single gene mutations. 
The Office of Rare Diseases identifies nearly 7,000 rare 
diseases and many of these are genetic in origin, often arising 
from single mutations. The ORD estimates that nearly 30 million 
Americans are affected by rare diseases. More than half of rare 
diseases affect children.
    Concomitant with advances in genetic diagnoses, there are 
parallel leaps in genome editing. CRISPR/Cas9 represents a 
significant advance for genome editing. Because of the co-
development of gene editing and stem cell biology, there is 
significant potential clinical application. Induced pluripotent 
human stem cells can be made from blood, skin, and other mature 
human cells. For my field, cardiology, skin cells can actually 
form beating heart-like cells in a dish allowing us to discern 
how mutations cause disease and letting us test how to correct 
these diseases. The human population is not placed at risk by 
these experiments in cells and it seems fair to say that the 
human population would actually be harmed by not doing these 
experiments since the research offers the potent opportunity to 
improve human health.
    Stem cells of the bone marrow, muscle, skin, and other 
organs can be isolated and edited. With these methods, it would 
be possible to cure sickle cell anemia or Duchenne muscular 
dystrophy. In mice, CRISPR/Cas9 mediated correction of 
fertilized eggs corrected the defect for Duchenne muscular 
dystrophy. The method, while imperfect, was associated with a 
remarkably high correction rate.
    Recently, a group of distinguished scientists called for 
careful consideration of gene editing in fertilized oocytes 
fearing the potential for germline gene editing and ultimately 
human eugenics. These discussions were enhanced and prompted by 
the recent report which we've heard about from Liang, et al., 
which described the efforts using CRISPR/Cas9 in fertilized 
oocytes.
    A regulatory framework for gene editing should encompass 
several key points. It should permit research to optimize and 
improve CRISPR/Cas9 and related technologies. It should permit 
in vitro and cell-based gene editing technologies, including 
those in embryonic and induced pluripotent stem cells. It 
should permit in vitro and cell-based editing with the intent 
to reintroduce cells into humans as a therapeutic measure for 
somatic cells. And it should permit the generation of gene-
edited animals for the purposes of scientific research. It may 
consider limiting or even prohibiting gene editing under the 
circumstances where human transmission of gene-edited germ 
lines would occur.
    But would we ever really consider germline gene editing? So 
we should consider the scenario of pre-implantation genetic 
diagnosis, otherwise known as PGD. PGD is pursued by families 
to avoid transmitting genetic diseases. PGD involves in vitro 
fertilization coupled with genetic testing. PGD is not covered 
by insurance, and yet for some families, they make this choice. 
These may be families who are already struggling with caring 
for one disabled child and who cannot care for a second 
disabled child.
    PGD is a personal option and one that is made solely by 
parents and families. In principle, it is possible that the 
efficiency of genome editing will improve so that pre-
implantation genetic correction could accompany PGD. With this 
process, gene editing to correct and eliminate a genetic 
disease could become reality. While the temptation may be to 
ban or limit this possibility, we should do so only with 
caution. Parents of children with genetic disease express 
significant concern, responsibility, and often dismay for 
having passed on mutations to their children. A parent's desire 
to protect children is undeniable. As a society and as a 
nation, we protect children.
    It may be tempting and easiest to ban gene editing where 
germline transmission could occur, yet the justified use of 
this approach is certainly conceivable and may one day be 
appropriate.
    Thank you.
    [The prepared statement of Dr. McNally follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]   
        
    Chairwoman Comstock. Thank you.
    And Dr. Khan.

                 TESTIMONY OF DR. JEFFREY KAHN,

           PROFESSOR OF BIOETHICS AND PUBLIC POLICY;

         DEPUTY DIRECTOR FOR POLICY AND ADMINISTRATION,

                 BERMAN INSTITUTE OF BIOETHICS,

                    JOHNS HOPKINS UNIVERSITY

    Dr. Kahn. Thank you. Chairwoman Comstock, Chairman Smith, 
and Ranking Member Lipinski, thank you for the opportunity to 
testify today on this timely and vitally important subject.
    As you heard in the introductions, I am a Professor of 
Bioethics in Public Policy at the Johns Hopkins Berman 
Institute of Bioethics in Baltimore.
    Also relevant to my comments today, I am also currently 
Chair of an Institute of Medicine Consensus Study commissioned 
by the FDA on ethical and social policy considerations of novel 
techniques for prevention of mitochondrial transmission in 
women to their offspring. Given that this study is considering 
issues related to the topic of today's hearing and the work of 
that committee is ongoing, I will restrict my comments to 
general observations and an overview of ethical and policy 
landscape related to gene editing.
    I'll focus my comments on three main topics: first, policy 
history and related areas of science and biomedical research; 
second, ethical issues raised by gene editing technologies; and 
third, relevant existing ethical frameworks and approaches to 
oversight.
    The relevant policy history started in 1975, and we heard 
some mention of this earlier, with the Asilomar Conference on 
recombinant DNA molecules whose summary statement focused on 
containment of the risks of creating and working with 
genetically modified organisms. And with the admonition to 
avoid experiments that ``pose such serious dangers that their 
performance should not be undertaken at this time,'' along with 
a call for continuing reassessment of issues arising in light 
of new knowledge gained with the experience of the then-new 
genetic technologies. And we've heard from Dr. Dzau that the 
National Academies are effectively taking on the same role 40 
years later.
    These voluntary suggestions that came from the Asilomar 
summary gave way to more robust oversight as the use of genetic 
technologies became more refined and with the initial attempts 
to treat disease in humans. It should be noted that that's 40 
years ago, and in the current environment, it would be very 
difficult for voluntary statements from a collection of even 
esteemed U.S. scientists to prevent research from going forward 
internationally, as we've seen already with the publication of 
the Chinese laboratory experiment.
    The ethical issues posed by gene editing and related 
technologies for modifying human DNA fall into three general 
categories of concern: first, the implications of the 
modification of germline DNA, and we've heard some about that 
already; second, the implications of interfering in processes 
that should be off-limits to humans. Sometimes those are sort 
of generally termed ``playing God,'' and that's problematic in 
some people's minds; and third, the potential for selection or 
introduction of traits for other than treatment or avoidance of 
disease, such as physical or behavioral traits or even 
enhancements.
    The focus of much ethical analysis in the application of 
manipulation of genetic information in humans has been on 
changes that affect the germline, that is changes that are 
heritable and therefore able to be passed on to future 
generations of individuals. The basis of these concerns relate 
to the uncertainty of the effects of genetic notification, the 
inability to ``undo'' unintended genetic changes or limit their 
effects, and the risks of passing on such unintended changes 
and their consequences to future generations. And that would of 
course go on forever.
    These ethical concerns have been addressed through a range 
of approaches in order to limit certain types of research or to 
provide prospective oversight prior to particular proposals 
being undertaken. We heard from Dr. Dzau that there are 
restrictions on federal funding of research that involves human 
embryos. Privately funded research is not affected by these 
restrictions, though the convention is that research on embryos 
should take place no later than 14 days after fertilization, 
and that's a limit also accepted by most countries engaged in 
research on human embryos. I should also note that there seems 
to be growing agreement that research should be restricted to 
nonviable human embryos.
    There are a number of institution-level oversight 
mechanisms that will apply to gene editing. That includes 
Institutional Biosafety Committees, which are charged with 
overseeing research with recombinant or synthetic nucleic acid 
molecules; Institutional Stem Cell Research Oversight 
Committees, which are charged with, as their name implies, 
research on human embryonic stem cells and related areas of 
research. And as the technologies are introduced into human 
subjects, Institutional Review Boards will be charged with 
overseeing any potential use on humans.
    Lastly, there's a role to be played by the scientific 
publication community. They have--journal publishers have an 
increasingly important role to play in setting and enforcing 
standards of behavior within the scientific community since 
publication of findings in the peer-reviewed scientific 
literature signifies the endorsement of the community of 
researchers.
    Journals also play an additional critical role with 
requirements on the ethics standards being respected, 
assurances authors' contributions are duly noted, and that 
human subjects are protected. So there's a role to be played on 
the part of the journal publication process that will restrict 
the potential of any unethical research going forward.
    Let me conclude by saying the United States has played a 
leadership role in this area in recombinant DNA and has the 
opportunity to do so going forward. There will be gaps 
identified in the process that the National Academies has set 
out on that need to be identified and addressed, and it is an 
appropriate time to consider what those ought to be.
    Thank you.
    [The prepared statement of Dr. Kahn follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
        
    Chairwoman Comstock. I thank the witnesses for their very 
interesting testimony.
    And now I remind Members that Committee rules limit 
questioning for five minutes.
    And the Chair--as Chair, I recognize myself for five 
minutes.
    So I wanted to hear a little bit more, Dr. Doudna, from 
what you mentioned where you had initiated the discussion with 
the scientists in California and bioethicists and others to 
address this. Some of the key--and maybe expand a little bit 
more on some of the key issues you found and how we avoid maybe 
where China might go and how we do define those boundaries and 
kind of what some of the concerns were that were raised. And if 
anyone else would like to address that, too, but I thought I'd 
start with you and maybe expounding a little bit more on this 
group.
    Dr. Doudna. Sure, thank you.
    I think, you know, what was interesting to me in that 
conversation was that it was a wide-ranging discussion that 
started with sort of the--maybe the way that we've been 
discussing this technology so far here in the Committee and 
eventually got to a point where, as somebody around the table 
said, you know, there may come a time when we would consider it 
unethical not to do editing in the germline for certain kinds 
of applications such as some of the things that Dr. McNally 
mentioned.
    So I think that it's very important to appreciate that, you 
know, this is a powerful technology that is--you know, we're 
sort of looking at it here from the perspective of safety and 
ethical concerns but I think they're also--you know, that can 
be turned around. And that was something that came out of our 
discussion in California that I found very interesting.
    And the other thing that we discussed was the fact that, 
you know, this technology, unlike previous technologies for 
genome editing, is very simple to employ relatively. I mean 
it's something that, you know, people that have expertise in 
molecular biology can fairly readily use in their laboratories. 
So I think, you know, the reality is that it would be very hard 
to really put regulations on this in terms of research 
applications.
    And I think that means that we just have to be thoughtful 
about providing leadership in terms of the--you know, the--as 
Dr. Kahn was saying, in terms of the way papers are published 
and reviewed among scientists so that, you know, the scientific 
community helps to provide the kind of direction and, you know, 
vision for the way this should move forward that hopefully will 
be respected by many others. But I think the reality is that 
it--you know, it is a technology that's just very widely 
available now and is being employed worldwide.
    Dr. Dzau. I'd like to echo what Dr. Doudna said. I think we 
at the Academies feel very privileged to be asked by the 
scientists--Ralph Cicerone, the President of National Academy 
of Science, myself, got lots of phone calls and emails to say, 
you know, the Academy really should take this on because we 
have done so in the past, as you heard, during the time of 
recombinant DNA, Asilomar conference, the human cloning, and 
also the whole issue of stem cell guidelines, as well as 
mapping the human genome. So we have to do this.
    I think that in the context of what's being discussed, it's 
really important that we have people from all different 
disciplines, not only scientists but ethicists, legal experts, 
medical physicians, and others all engage in this discussion 
because at the end of the day what we want to do is to find 
what appears to be the consensus of what the right thing to do 
is for our country and so we look forward to working on this.
    Dr. Kahn. So I would just like to echo what you've heard 
from my colleagues on the panel and add a little bit to expand 
on what I said about publication. It's difficult for the 
scientific community to identify any one thing that it can take 
on in an international way, but scientific publication knows no 
borders. And there have been calls now for--before publication 
of any gene editing research that there be a disclosure of the 
ethics review that that research underwent. That did not happen 
so far as we know with the Chinese research that was recently 
published, and in fact, the reports have been about Science and 
Nature rejected that paper when they considered it and at least 
in part on ethics grounds. So that would be one way to create 
some--a framing of what would be acceptable as determined by 
the scientific community itself through the peer review 
process.
    Chairwoman Comstock. Thank you. And, Dr. McNally, did you 
have anything to add? I have a few more seconds before I turn--
--
    Dr. McNally. I would also remember that through this 
process it's not just the scientists and the physicians and the 
regulatory bodies but to also remember the patient advocacy 
groups and the patients themselves. They're going to be the 
loudest voice in this process and we need to hear what they 
have to say.
    Chairwoman Comstock. Thank you. I appreciate it.
    Now I recognize Mr. Lipinski for five minutes.
    Mr. Lipinski. Thank you.
    Before I follow up, I just want to make sure we're more 
clear on this when we get into the subject.
    Dr. Doudna, you and the group that have met, you've called 
for a temporary moratorium on the use of the technologies on 
human embryos. Is that correct? And what led you to that and 
are there particular milestones and discussions of the science 
that you're working towards and then see ending the moratorium? 
And I want to get the other panelists to comment on that.
    Dr. Doudna. So I would say what led us to that initial 
meeting in California was the appreciation that this technology 
would likely, you know, be functional in the human germline, 
and furthermore, that it was possible that people could do this 
fairly easily and perhaps working in jurisdictions where there 
would not be regulatory oversight of such experiments. And what 
was interesting was that at that meeting we actually heard 
about the work that was subsequently published for the Chinese 
group, so it became apparent that, you know, the subject of 
that conversation was very timely.
    I think that, you know, going forward we really have to 
appreciate the difficulty in putting in place regulations that 
will be, you know, followed internationally. On the other hand, 
providing oversight and leadership in--by respected scientists 
in the United States and with our international partners I 
think will be a very important thing to do and that's really 
what we wanted to achieve at that meeting was how to proceed 
with that sort of approach. And I think now having the National 
Academies involved in organizing larger meetings around this 
issue is a very desirable outcome.
    Mr. Lipinski. Does anyone else want to comment on a 
temporary moratorium on human embryos?
    Dr. Dzau. So the National Academies' position is that we 
need to be thoughtful, comprehensive, scientifically driven, 
and independent so we are convening those bodies and obviously 
supporting the idea that we should be very cautious. We have 
not actually taken the position per se because until our work 
is done, you know, we would be looking at what the most 
objective way to approach this when our work is done.
    So I think some of these meetings are critically important 
and important in the sense that we need international 
scientists to be involved with this conversation as well. We 
have in fact on our advisory board scientists from the Royal 
Society in London, scientists from the Chinese Academy of 
Science involved with this conversation because it's really 
critical to consider all aspects.
    As Dr. McNally pointed out, you know, if you're using 
certain conditions, I think we have no doubt that this would be 
enormously helpful to patients and to mankind. The question is 
what are the areas we need to be concerned about?
    That being said, I think technology is still early because 
even when you look at the Chinese publication, they were 
looking at incomplete editing and many other issues that 
research has to go forward to understand the safety, the off-
target effects, and the efficacy before human application. So 
that's our position.
    Mr. Lipinski. Okay. Dr. McNally?
    Dr. McNally. I would like to echo what Dr. Dzau just said 
which is that I appreciate very much the leadership by Dr. 
Doudna and the group of scientists that got together but I 
think the IOM convening an international effort to really look 
at what the possibilities are I would think it's reasonable to 
wait until we learn what that outcome is. It's important to 
have a careful and to have an international look at what the 
best recommendations really are at this point in time.
    Mr. Lipinski. Dr. Khan?
    Dr. Kahn. Just very briefly, the kind of work people are 
talking about on an international level is really critical in 
establishing principles and guidelines for how to move forward. 
I think moratoria--blanket moratoria are probably not what we 
need but figuring out when we can go forward and how and under 
what kind of restrictions. So certainly in vitro only and 
probably, you know, for a very long time until it's determined 
that the milestones have been met in order to move forward into 
humans. So that's the good work that entities like the 
Academies can engage in and I think critical at the 
international level.
    Mr. Lipinski. Thank you all very much. It's--yeah, I think 
there are very difficult ethical questions we need to deal with 
here and I know myself I certainly don't know enough about the 
technologies, some of the specifics there, and I think that 
makes--certainly makes a difference. And then we do get to a 
question that Dr. McNally said there are things that we 
certainly want to cure and then the question always is how far 
do we take this? But we're certainly not going to solve that 
here so thank you very much.
    Chairwoman Comstock. Thank you.
    Now I recognize Mr. Moolenaar for five minutes.
    Mr. Moolenaar. Thank you, Madam Chair.
    And thank you for your presentations today and my 
compliments to you on the work you're doing. It's very 
sophisticated and it seems that it has tremendous potential for 
good.
    On the area of ethics of this I wanted to just explore a 
few different areas. Dr. Khan, I wanted to start with you. When 
you're advising on the ethics of a new genetic technology, are 
there certain ethical lines that should never be crossed, and 
if so, how are those lines drawn?
    Dr. Kahn. That's a great question and how much time do we 
have I guess is going to be part of my answer. But of course 
those of the first kind of questions that we ask.
    I will say there has been a long-standing line that has not 
been crossed and that is modification of the human germline. 
That has held for decades since the beginning of recombinant 
DNA technology was available and begun to be implemented.
    One thing that none of us have actually talked about is the 
recombinant DNA Advisory Committee, which is a committee of the 
advisory to the Director of the NIH, which has in its 
guidelines a statement that they will not consider any proposed 
research that modifies the human germline. So it's effectively 
a prohibition from that research going forward. Now, it only 
applies to research that is subject to the oversight of that 
committee, but that has been a bright line.
    The other thing which we sort of skirted around but haven't 
maybe set explicitly is that when there's such uncertainty 
about the risk and the outcomes, we go slowly. So that's the 
kind of a soft answer to your question, a mushy one, but I 
think that's an important principle to--just to articulate in a 
way that's explicit. When we know it's time to go forward is a 
harder question but we always start slowly, especially when 
we're talking about potentials for modifying humans.
    Mr. Moolenaar. Thank you.
    And then, Dr. Doudna, you're one of the new developers of 
this new technology, and what was it that first sparked your 
concern over the ethics of its use, whether it was in China or 
when did you start being concerned about that?
    Dr. Doudna. I guess I realized the potential for this 
technology to operate in the germline first when scientists 
began to do experiments of that nature in animal models of 
disease, including mice and rats, and then it really came home 
to me in--it was last year--may be almost a year-and-a-half ago 
that a group again from China published a paper in which they 
had modified the germline of monkeys and made genetically 
modified monkeys. And that actual monkey model is used very 
commonly for studying human disease and so it seemed very 
likely at that point that there was no reason to think the 
technology wouldn't also work in the human germline.
    So, you know, I think we've seen now in the scientific 
community that this technology is very democratic in the sense 
that it works across many different types of cells. It doesn't 
seem to be limited to a particular system.
    Mr. Moolenaar. And what role does--you know, I guess one of 
the things that occur to me is the whole area of consent. What 
role does that play in this process when someone is giving 
consent or not?
    Dr. Doudna. Are you directing that question to me?
    Mr. Moolenaar. Yeah--actually, all four.
    Dr. Doudna. You know, I would--maybe I would defer that to 
Dr. McNally.
    Dr. McNally. Well, as a member of an IRB for about 15 
years, that's actually--you know, right now, consent in a 
research study in that case would be the parent; the mother 
would be the person providing consent. If a study were to go 
forward right now, that's who would be providing consent 
because it would be her materials that were being used for that 
purpose. So there isn't--from the standpoint of human subjects 
strictly talking like Institutional Review Boards, a fertilized 
egg is not an individual that provides consent and also even--
you know, as a--if it were a child, a parent provides consent 
for that if that answers your question.
    Mr. Moolenaar. Yeah. Thank you.
    Dr. Kahn. So it's--I would sort of ask consent from whom 
and for what? So you've heard a version of the answer to that 
question. If we're talking about the donor of the materials 
that would be researched upon, that's one set of questions. If 
we're talking about modifying an embryo that might one day be 
implanted into a woman's body and developed into a child where 
we have a very difficult conceptual problem. How do we think 
about consent on behalf of somebody who's not yet been born? 
And so those are the really interesting ethical questions that 
we will need to confront. We're certainly nowhere near thinking 
about doing that kind of application, but those are the kinds 
of questions that need to be identified, articulated, and 
addressed in efforts that are like the ones the Academies are 
taking on.
    Mr. Moolenaar. I would agree with that. Thank you.
    Chairwoman Comstock. Thank you.
    And now I recognize Mr. Tonko for five minutes.
    Mr. Tonko. Thank you, Madam Chair.
    This hearing shines a light on a difficult but indeed 
important issue and I appreciate my colleagues' focus on the 
ethical and legal issues surrounding this new technology.
    I'm also assured to see that experts in the medical and 
scientific community are coming together to debate this issue 
and to discuss potential policy implications. However, as we 
explore the boundaries of what science is capable of and what 
is ethical and what should be legal, we should also take a 
moment to appreciate just how remarkable these advances are. We 
recognize that these new gene editing technologies, including 
CRISPR, are the outgrowth of decades of fundamental research 
supported by federal agencies, including the National Science 
Foundation.
    So to Dr. Doudna and Dr. McNally, could you please speak to 
the importance of federal investments in fundamental research, 
especially the need to support research that may not have any 
known commercial application at the time?
    Dr. McNally. Again, how much time do we have?
    Mr. Tonko. Well, the Reader's Digest version.
    Dr. McNally. I think everybody sitting here at this table 
can say it cannot be overstated how important the federal 
investment is for research. There are many people who would 
love to see a lot of research funded in the private sector but 
there are certain aspects of particularly fundamental research 
that will never be covered in the private sector. Sequencing 
the genome is a great example of that.
    And we can't move forward without that federal investment 
and I think all of us here would say, you know, it's been 
fairly tough times in the last few years with what's happened 
with budgets and what's happened with research and watching the 
effect that that has had on the scientific community here in 
the United States where we have actually seen the size and 
shape of the scientific community shrink in the last few years, 
especially when we look across the world and we see it growing 
elsewhere. So, yes, federally funded research is absolutely 
essential for these types of basic observations.
    Dr. Doudna. Right, so I echo everything that my colleague 
Dr. McNally just stated and I want to also add that, you know, 
there's a tremendous opportunity for the United States to 
invest in basic science. I mean I think traditionally our 
country is been a leader in science partly because we have 
invested in science that was, you know, curiosity-driven 
research. It was not necessarily targeted on curing certain 
diseases, and I think that we've seen again and again, 
especially I would say with regard to technologies, they tend 
to come from unexpected types of projects such as the CRISPR 
system is a great example of that but there are many others. 
And I think also it's important to appreciate that 
commercially, you know, these things then have big implications 
in terms of companies being able to take over and, you know, 
develop technologies that are discovered in academic 
laboratories but then apply them in all sorts of different 
ways.
    Mr. Tonko. Dr. Dzau, I think you wanted to respond to that, 
too?
    Dr. Dzau. Well, I totally agree with my colleagues and 
they're particularly emphasizing support for basic research 
because if you think about this technology, it was done on 
bacteria, and without thinking of application human and look 
where it is today. And we can count so many important 
breakthroughs that come this way. So the ability to support 
fundamental basic research is critically important.
    Also, I think along the issues about saving human lives, 
creating jobs, is our global competitiveness situation, we are 
truly concerned that we don't continue this level of investment 
that the United States becomes less competitive than many other 
countries which are investing heavily into basic and 
translational research.
    Mr. Tonko. Thank you.
    Dr. McNally, in your testimony you mentioned how these 
technologies may be able to be used in somatic or mature cells 
to treat and potentially cure diseases such as sickle cell 
anemia and muscular dystrophy. Can you please elaborate on this 
possibility and what is that range of therapies and cures that 
we might only imagine?
    Dr. McNally. I think it's widely anticipated that sickle 
cell will be one of the first things that's cured by this where 
you could take a cell out because it's a bone marrow cell. You 
could correct it with CRISPR/Cas and return that person's bone 
marrow cells so it's not a transplant situation; you're 
returning their own cells to them. And that's ongoing right now 
and I anticipate that we will see that.
    For my field I work in muscle diseases. Duchenne muscular 
dystrophy is a very challenging, challenging area and right now 
we're looking at technologies where we're taking small 
antisense compounds where we would have to treat that 
individual for a lifetime every day with those compounds. 
Again, if we could get the cells, correct them, and reinsert 
them back in, that would be a one-time treatment and a lifetime 
treatment for that individual. So I think we're seeing a few 
examples where it's definitely heading that direction.
    Mr. Tonko. Thank you so much.
    Madam Chair, I yield back.
    Chairwoman Comstock. Thank you.
    I now recognize Mr. Palmer for five minutes.
    Mr. Palmer. Thank you, Madam Chair.
    Several folks have mentioned the research on stem cell. Dr. 
Tim Townes at UAB is a very good friend of mind and doing 
world-class research in that area.
    I've got just a few questions. Dr. Dzau and Dr. Kahn, the 
United States and Europe have often disagreed on regulations 
and policies and other areas of biotechnology, for example, 
genetically modified organisms. How does that impact 
international scientific cooperation? And do you anticipate a 
similar challenge in human gene editing?
    Dr. Dzau. Well, the intention is that when you get 
scientists, regulators, ethicists together from different 
countries, I think responsible individuals would begin to talk 
about what would be responsible behavior. That I believe is the 
starting point. And I do agree with you that we have some 
differences in the regulation. But I think overall in the issue 
we're talking about today, which is the application in germline 
gene editing, I have a sneaking suspicion--although I don't 
want to fully predict this until the work is done--there's 
great agreement about the concern about creating successive 
generations of individuals whose genes have been altered. So I 
have a feeling that we actually will get agreement if not 
harmonization of many of these thoughts, and certainly it's our 
hope that we will reach that in our initiative.
    Mr. Palmer. There are current regulations that prohibit 
federal funding for human--for research on human embryos and 
the FDA requires--must issue an investigational new drug 
application before a biological product may be used in humans. 
Do you think these kind of safeguards are adequate to prevent 
the kind of experiments that we're concerned about?
    Dr. Dzau. I'd like Jeff to answer this as well because, as 
you heard, he's in the midst of leading one of our initiatives 
on mitochondria DNA replacement.
    Mr. Palmer. Um-hum.
    Dr. Dzau. But I think we very much look into what is the 
right regulatory framework particularly for issues like this, 
and I would say that, you know--and because he would have an 
opinion but my feeling is that this work needs to be done to 
get better clarity about when and how we would regulate some of 
these areas.
    Mr. Palmer. Dr. Kahn?
    Dr. Kahn. Thank you. In answer to your question, I think 
that the FDA will play a critical role although it will only 
play its role towards the end of the story so the very basic 
research will be done prior to anybody thinking about an IND 
application or introducing it into a therapeutic context. And 
so we need to think about the entire translational pipeline as 
it were and all of the issues that will arise along the way and 
make sure that we have appropriate oversight for each of them.
    The FDA is clearly thinking about the issues that you are 
and are trying to figure out how their framework ought to 
apply.
    So first--for first in human applications effectively, and 
then once something is licensed, how to control its use and 
dispersal. So one of the things that we all worry about is the 
so-called off-label use of the new technology. So even though 
the FDA may approve it only for a limited purpose, once it's 
licensed, it's hard to control. The FDA has some new tools that 
may actually make them more feasible to do and I think as we 
get closer to the kinds of technology were discussing entering 
the therapeutic marketplace that there will be stronger 
safeguards in place.
    Mr. Palmer. Well, with these safeguards--and staying on 
that line of thought--is there any worry that if the United 
States doesn't use this research that we could fall behind our 
international competition and, you know, could new regulations 
of this technology in the United States put our researchers at 
a disadvantage or cause them to move their research overseas, 
Dr. Kahn?
    Dr. Kahn. I'll let Dr. Dzau and others speak to this, too, 
but there always is talk about that and of course it became a 
bigger issue when the stem cell research--human embryonic stem 
cell research field really began to grow. And there are now 
international stem cell research societies which try to address 
issues that are clearly not governed by borders. And what we 
don't want of course is to have people who are doing the best 
science in the world think about leaving this country because 
it's easier to do elsewhere. So we need appropriate controls 
but not those that squelch the science. Finding that sweet spot 
of course is the challenge.
    Mr. Palmer. One last question and you can follow up on that 
as you answer this also, but do you believe that we'll be able 
to get China and the United Kingdom and these other nations to 
work together to influence change and Europe to adopt similar 
standards--similar safeguards?
    Dr. Kahn. Maybe Dr. Dzau can speak to that if that's okay.
    Dr. Dzau. Well, we're starting with getting the major 
science academies involved so we have the National Academy of 
Science and Medicine in the United States; we have the Chinese 
Academy of Science where you would think that they have 
tremendous influence on the way the conduct of science is being 
carried out. We have the Royal Society and we intend to include 
many international bodies.
    So I think the starting point clearly is with the 
scientists saying what would be the right thing to do. One can 
imagine that we may have to escalate this conversation further 
depending on our findings.
    I just want to point out the question that you asked, which 
is the regulation aspect of this. In fact, you know, we do need 
a lot more clarity in this country. We haven't talked about the 
use of gene editing in nonhuman cells, plants, insects, and 
those changes are--that science is really ongoing. We are 
producing possibly new species that would turn around much 
faster because of a much shorter cycle time, reproductive time. 
So that regulation also has to come in to say what in fact is 
considered safe and what's considered as environmentally sound. 
And in fact the National Academies is also looking at a study 
looking at this issue. And as you know, in this country the 
USDA and FDA are involved with animal and plant regulations. So 
we're also trying to give the right recommendation to fortify 
our regulatory processes.
    The final question you asked earlier about what the right 
thing to do is I think from our perspective at the National 
Academies I think our first issue is human protection and doing 
the right thing for society. I understand of course the 
potential loss of scientists, et cetera, but I do think that we 
have to actually take the high road to say what's right for us 
first. And I have a feeling everything else would follow and 
fall in the right place.
    Mr. Palmer. Thank you.
    Thank you, Madam Chair.
    Chairwoman Comstock. And I now recognize Mr. Swalwell for 
five minutes.
    Mr. Swalwell. Thank you, Madam Chair, and thank you to our 
panelists.
    My first question relates to something that a number of the 
Members have brought up, which is it seems that America and our 
investments in federally funded research have been in decline, 
and as a result, our successes have been in decline and our 
ability to attract and recruit and retain some of the best and 
brightest scientists may be in decline. So let's just for 
argument's sake go back to 1995. I was 14 years old. And the 
Human Genome Project was just starting to get off the ground 
and many great results came out of that. That was 20 years ago. 
Would each witness just say more or less has America and its--
have you seen the investments that we've made as far as 
federally funded research, has that made America more or less 
exceptional in this field? So just tell me more or less. And 
I'll start with Dr. Dzau, just one word.
    Dr. Dzau. More.
    Dr. Doudna. More.
    Dr. McNally. More.
    Dr. Kahn. More.
    Mr. Swalwell. So I'm confused because each of you has said 
that our investments have been on the decline and so you're 
telling me that we actually have made more investments since 
1995 and you believe we are more exceptional now in these 
fields?
    Dr. McNally. You picked 1995.
    Mr. Swalwell. But comparing----
    Dr. McNally. If you said 1995----
    Mr. Swalwell. Sure.
    Dr. McNally. --to 2005, we would say more. If you would say 
2005 to 2015 we would all say less.
    Mr. Swalwell. Is that right, less, Dr. Dzau?
    Dr. Dzau. [Nonverbal response. Nodded in the affirmative.]
    Mr. Swalwell. Dr. Doudna?
    Dr. Doudna. [Nonverbal response. Nodded in the 
affirmative.]
    Mr. Swalwell. Dr. McNally?
    Dr. McNally. [Nonverbal response. Nodded in the 
affirmative.]
    Mr. Swalwell. Dr. Kahn?
    Dr. Kahn. [Nonverbal response. Nodded in the affirmative.]
    Mr. Swalwell. Okay. So that was my question. You would 
agree that we have become less exceptional in the field of 
genetic engineering as far as it relates to human DNA since 
1995, that we've been on the decline?
    Dr. McNally. 2005.
    Mr. Swalwell. 2005 is the----
    Dr. McNally. Yes.
    Mr. Swalwell. --point, Dr. McNally?
    Dr. McNally. Yeah.
    Mr. Swalwell. So what's exciting about this research and 
this field is the potential for us to conquer diseases before 
they conquer us. And I look at the example of Huntington's 
disease, which affects anywhere from 30,000 to 200,000 people, 
and it's a disease that is so cruel it steals your memory and 
affects your muscular system.
    And I'm just wondering, maybe if Dr. Doudna can tell us and 
have others weigh in, what can the United States do 
specifically to take leadership in this area if we have the 
appropriate funding so that we can conquer these diseases?
    Dr. Doudna. So I think I know, what--again sort of maybe 
echoing something that I mentioned earlier and that has been 
discussed here, I think, you know, the United States has been a 
real leader in basic research for a while and all of us are 
concerned that we see that edge slipping away over time. And so 
I think that, you know, the investment in fundamental research 
that will allow scientists to understand, for example, genome 
engineering technology like we're discussing today, how does it 
operate, how can we deliver it to patients, how do we ensure 
that it's operating as we intend and not creating unintended 
consequences, that it's safe, that it's effective.
    All of those lines of research are going to require, I 
would say, a combination of efforts by people like me that do 
basic research and people like my colleagues who are medical 
doctors and think about clinical issues. We need to be putting 
our efforts together and that has to be I think supported by 
federal funds.
    Mr. Swalwell. And how can we tell the story to the American 
people who look to us as a Congress to make the decisions when 
it comes to funding with so many competing priorities? How can 
we tell them that something--like $1 invested in basic research 
where you may not be able to tell us what disease you're going 
to be able to cure 10 to 15 years from now but there's still--
the taxpayer is looking to us to, you know, hold accountable 
the funding. Like how can we better tell the stories of the 
science community about what we could see from this down the 
road and how we could truly, you know, attack and cure some of 
these diseases? I think that's probably one of the biggest 
challenges. And maybe Dr. McNally would want to answer.
    Dr. McNally. Yeah, I mean we've seen incredible advances 
that have come out of basic research, and I'll talk about my 
field, which is cardiology. It was out of basic research of 
understanding the LDL receptor that led to statins, the drugs 
that probably a lot of people in this room take, and we've seen 
a direct translation to a reduction in the rate of heart 
attacks. I mean it's a very different world than what it was 
when I first heard of my training 20 years ago. We don't see 
acute heart attacks like we used to and that came as an outcome 
of basic research not that many years ago.
    So we can tell the same story for heart disease. We can 
tell the same story for many cancers. Cancer and heart disease 
are the major things that kill people so we've made huge 
headway in that.
    Mr. Swalwell. Great, thank you. And, you know, I know every 
Member up here has thousands of people in their district who go 
to bed on their knees praying that people like you will make 
discoveries that will make them or their relatives live 
healthier lives. So thank you for what you're doing and 
hopefully we can do the right thing here and better fund your 
initiatives.
    And I yield back.
    Chairwoman Comstock. Thank you.
    And I now recognize Mr. Westerman for five minutes.
    Mr. Westerman. Thank you, Madam Chair, and thank you, 
panel, for your invigorating testimony.
    Dr. Dzau, what's your anticipated timeline for the 
initiative on human gene editing?
    Dr. Dzau. We are convening an international summit in the 
fall--late fall and that should be a--I think really an 
important meeting that will get together international 
scientists. As you heard from our previous experience at 
Asilomar in the 1970s, you know, one would engage in the 
discussions that Dr. Doudna and McNally and Kahn put forth, and 
hopefully the findings of that meeting will be published very 
shortly after that.
    Perhaps equally important is a concurrent deep-dive study 
which we're conducting that would involve analysis, research, 
the assessment of risk/benefits, as well as a regulatory 
framework and ethical issues, and that usually--what we call a 
consensus report would take as long as about a year, although 
we're hoping that we would try to move it faster for exactly 
the reasons that we've been talking about. Such a report with 
very specific recommendations which we're willing to put 
forward under both public hearing and also closed discussions 
will be available to you, to the Nation, and to many others. So 
I could say the time frame is late fall to sometime next year 
but hopefully as early as we possibly can to put out that 
report.
    Mr. Westerman. So how great are your concerns that the 
initiatives may not be able to keep up with the breakneck speed 
of the technology as it moves forward?
    Dr. Dzau. This is exactly why we need a summit, a meeting 
first, where key scientists--and we're going to engage a large 
number--will discuss about what would be considered as good 
conduct, good oversight, understanding risk, et cetera, so that 
the scientific community which is really driving most of this 
research understands those issues and have in general 
agreement.
    We also believe of course in our study itself that becomes 
a definitive document by which you and others can be informed 
to say what are the right decisions based on consensus.
    Mr. Westerman. So you said the scientific community is 
driving the research. As the National Academies' work on the 
new initiatives for building the framework, do you believe the 
scientific community will embrace and voluntarily follow the 
guidelines or will there need to be regulations or laws put in 
place?
    Dr. Dzau. Well, you know, as you already heard, I think 
there are many responsible scientists, particularly the ones 
who are leading this field, who feel already that we should, 
you know, put a moratorium and not slow it down. So I do 
believe that already going into this meeting, although there 
may be many different opinions, there's probably general 
agreement that we need to slow down this area until we have a 
much clearer point of view about where we should be going and 
the clarity in terms of regulation.
    The problem I'm concerned about is that we rush into this 
too fast. We don't really want to and it's such an important 
issue. We've got to be very thoughtful. That being said, we 
understand the time urgency of the issue--situation.
    Mr. Westerman. All right. And the advisory group to the 
initiative that was named yesterday, it includes scientists and 
researchers from China and the United Kingdom. Do you believe 
these participants will help influence Asia and Europe to adopt 
similar standards?
    Dr. Dzau. We certainly hope so, and in fact I was on the 
advisory group. Our intention is that in an international 
summit we include a lot more scientists from Asia and every 
part of the world, Europe, et cetera, to be part of this 
discussion.
    You know, it's interesting when we think back on U.S.'s 
position. You know, when the Asilomar Conference come about, 
the United States was the main show in town about technology, 
so among the U.S. scientists, you can imagine there's 
agreements, you know, so there's a general way of saying, you 
know, what do we do next? Here, we really need to include 
international scientists.
    Mr. Westerman. So what capacity and infrastructure does the 
Chinese Government have in place for regulating human 
scientific research?
    Dr. Dzau. You know, I'm not that familiar with the 
regulation at this point in China. As you heard from my 
colleague, Dr. Kahn, there is some speculation over what's 
there and what's not there. I think we would hope that that 
meeting will bring out with clarity what each country's 
position is, what's the regulatory position, what's the 
scientific position so that we can all come together and 
examine this together.
    Mr. Westerman. Thank you, and I believe I'm out of time, 
Madam Chair.
    Chairwoman Comstock. All right. Thank you.
    And I now recognize Mr. Foster.
    Mr. Foster. Thank you, Madam Chair.
    In addition to the international summit that you're having 
this fall it strikes me that a full-blown National Academies 
study may have merit to do a deeper dive into this. And so, Dr. 
Dzau, would a letter signed by Members of Congress, for 
example, help you in recruiting assistance for this sort of 
effort?
    Dr. Dzau. I think it would be outstanding in fact if we got 
a letter from Congress on this. We took this upon ourselves as 
a National Academy because that's what we do, and we know it's 
the right thing to do. We have the support of scientists and 
others to say go forward. But I think it would be tremendously 
impactful if Congress would provide us with that kind of 
support a mandate to go forward with this.
    Mr. Foster. Okay. Just a quick question, today what's the 
rough cost and time to get a mouse model with a specific 
genetic modification? Does anyone--just roughly within a factor 
of two--you know, is it $10,000 or $5,000 or----
    Dr. McNally. Yeah. Well, with CRISPR/Cas initiated, yeah, 
you'll probably decrease the cost in half and decrease the time 
in half. So if it was $25,000, it's probably closer to 10 now, 
and if it was a year, it's probably closer to 6 months. That's 
counting breeding time.
    Mr. Foster. Well, when--so one of the things slowing down 
the application of this is a lot of--sort of two classes of 
worries about potential dangers. The first is so-called off-
target effects where, in addition to the genetic modification 
you want, you get inadvertent modifications to the genome. And 
it's my understanding that the technology is evolving rapidly 
and that--I was just wondering if you--I'll ask you to go out 
on a limb I guess, and Dr. Doudna first, about--you know, if 
you just look at the rate of progress on this, what is the 
rough timescale where we can expect--where we might expect 
you'll be in a position that it could be used, you know, 
``safely'' on humans?
    Dr. Doudna. Well, I think one has to, you know, sort of 
distinguish what types of applications we're talking about. I 
think if we're considering application to treat a disease like 
sickle cell anemia where the editing could be done on cells 
that are taken out of the patient and then validated before 
they are reintroduced into the body, I feel that that is likely 
to happen, you know, the next year or two honestly. I think it 
will be very----
    Mr. Foster. Right, so no technological development there?
    Dr. Doudna. No, because I think we already have the ability 
to, you know, validate the correct sequencing--correct editing 
was done by DNA sequencing in that sort of a scenario. I think 
if you're talking about an application like, you know, we want 
to introduce the tool into a patient's body and where you want 
editing to occur in the body, then we're--that's further off. 
First of all, we don't have the--very good ways to introduce 
this into specific tissue types yet, and also we don't have 
good ways to validate that the correct type of editing was done 
without off-target effects, as you implied. But I think for any 
kinds of applications where we can do the validation outside of 
the body, that's going to move forward in the next year or two.
    Mr. Foster. Okay. And then if I raise the stakes further to 
germline editing, is that something that may just never happen? 
It may never be reliable enough? Or is it a reasonable guess 
that within the next five years that you'll be able to validate 
the germline editing, that it has taken place correctly and 
with high enough confidence that--
    Dr. Doudna. Well, I'm very interested to hear my 
colleagues' answer to that question but I guess my answer would 
be that it will depend on the way that research is enabled 
around, you know, that sort of application. I think if it's 
possible to do experiments in germ cells so that we can 
understand how this technology works, operates in those types 
of cells, then I think, you know--boy, it's always hard to put 
a timeline on things but, you know, certainly within a few 
years it'll probably be to the point where one could, you know, 
employ it for that sort of application. But I don't know if 
you--my colleagues would agree with that or not.
    Dr. McNally. I agree. I think that's a reasonable timeline, 
five, ten years if you had to guess.
    Mr. Foster. Yeah. I'm trying to, you know, get some idea--
--
    Dr. McNally. Yeah.
    Mr. Foster. --of what the response time from Congress and 
our society has to be for that.
    There's a second class of potential dangers having to do 
with just misunderstandings about what the effects of a 
specific genetic change will be on the characteristics of the 
adult organism. And, you know, over the spread of, you know, 
different things from simple conditions like sickle cell to 
complex things like, you know, personality, you know, what is 
your guess for the timescale that we're looking at there from 
right now to never?
    Dr. Doudna. My answer is certainly much longer. I think 
we--I think--and that--to me that's not limited by the genome 
editing technology as much as it's limited by our knowledge of 
the human genome.
    Mr. Foster. Okay. Thank you. My timer has gone down. I 
yield back.
    Mr. Moolenaar. [Presiding] Thank you. I now recognize Dr. 
Abraham.
    Mr. Abraham. Well, after I read you all's testimony last 
night I went on and read each of you all's bios. Your parents 
must be very proud.
    We all in this room I think understand the potential that 
this type of research can lead to not only in the human 
endeavors but in plant technology, curing world hunger. So it's 
applicable to so many aspects of humanity.
    And to--I think it was Dr.--your point, Dr. McNally, that 
it probably would be unethical if we have a child with sickle 
cell in the ALL and this therapy is available not to offer it.
    Dr. Doudna, I salute your intelligence for recognizing it 
even though I know you weren't particularly looking for the 
CRISPR/Cas9 technology that you recognized it. I compared it 
last night--I was reading--to Fleming discovering penicillin. 
He had been--I think maybe this CRISPR can save as millions of 
lives as penicillin has, so kudos to you guys for what you do.
    I guess the question I'm leading up to, the old adage if 
you get two doctors in a room, you get three different 
opinions, as we know very true. That's certainly on my end of 
the stick.
    Dr. Doudna, you published I think an article in a science 
magazine that you wanted this moratorium and, Dr. Dzau, you've 
been pushing for. Are you guys getting some pushback from 
members of your community that says, you know, no, we don't 
need a moratorium and let this thing go? Let this genie out of 
the bottle and don't put it back in?
    Dr. Doudna. Well, I can tell you what I'm seeing. I think 
that, you know, at around the same time that we published the 
perspective in Science magazine a related perspective was 
published in Nature magazine from a different group that 
actually called for I would say real moratorium even on 
research. So that group basically was advocating not proceeding 
with any kind of research on human germ cells using genome 
editing technology. And I just want to point out that in the 
group that I met with in January, we actually discussed that 
and felt that actually we--in our opinion research on those 
types of cells, appropriately regulated, should be enabled, 
just not clinical application.
    Mr. Abraham. Dr. Dzau, when--Dr. Kahn, when you had the--I 
think it was back in 1975 I was reading last night y'all had a 
recombinant DNA moratorium that you tried to put forth 
voluntarily. In the international community, was that followed? 
Has that been pretty much adhered to throughout the last few 
generations?
    Dr. Kahn. So a little bit before my time but it's a 
landmark in the area of science policy and then it was a 
voluntary moratorium instituted by the scientific community. 
The truth is we haven't seen anything like that since. It was a 
very important undertaking. And I think when the scientific 
community got together to talk about the implications of 
recombinant DNA technology at that time, they weren't sure 
whether the scientific community would follow what the----
    Mr. Abraham. Much like now.
    Dr. Kahn. Much like now. It's 40 years later and the 
scientific community is very different today than it was in 
1975.
    Mr. Abraham. For sure.
    Dr. Kahn. So we were undisputedly the leaders in the world 
of that science in 1975. As everybody has said, the technology 
now is much more widespread and much easier to implement, as 
Dr. Doudna has said, making it much more difficult for any one 
community of scientists to actually speak on behalf of the 
whole.
    Mr. Abraham. Well, we think--we can probably say 
realistically that America will lead in the discussion of the 
ethical and moral implications of this, but to follow up with 
your statement then, as Americans or as the United States----
    Dr. Kahn. Um-hum.
    Mr. Abraham. --scientific community, if we should see an 
element get outside the bounds, can we do anything?
    Dr. Kahn. Yeah, it's a great question. And as I said in my 
testimony and in my statement that you read and I reiterated 
today that the journal publication community has a very 
important role to play here. What--your research doesn't really 
mean much unless your peers have reviewed it, called it good, 
and then it gets published in a credible place. So that's a 
really important barrier. That doesn't mean people won't try to 
do things that aren't ethical and then try to get them 
published, but it's a--has a sort of strong inoculating feature 
I would say.
    The other thing that Dr. Doudna pointed out but I'll 
reiterate is that there's been a little bit of a disagreement 
in the scientific community on the topic of gene editing, about 
whether there should be a moratorium only on clinical 
application or on something more widespread. And so that's a 
healthy debate to have and it's great that we're having it and 
it's great that the Academies are bringing it out to the 
international level. So that's exactly the discussion we ought 
to be having.
    One last thing I'll say is that people around the world 
want to be part of the scientific community. There's a very 
strong incentive for them to behave, right, to follow the 
conventions which make them a legitimate member and I think we 
shouldn't underestimate the power of that. So, yeah, there 
might be fewer restrictions in other parts of the world but 
those people want to publish in Science and Nature just like 
American scientists do.
    Mr. Abraham. Yeah. Thank you.
    Mr. Chairman, I'll yield back.
    Mr. Moolenaar. Thank you.
    I now recognize Mr. Sherman.
    Mr. Sherman. Thank you, Mr. Chairman, for holding these 
hearings. This has been an area of intense interest on my part 
since the year 2000 when I went to the Floor and said that the 
most important decision we will make this century is whether 
our successor species is carbon-based or silicon-based, whether 
the new and intelligent species on this planet is the product 
of genetic engineering or the product of computer engineering.
    Some of you will remember I served on this Committee in the 
107th and 108th Congresses and this was pretty much my main 
reason for serving on the Science Committee.
    I should bring to the attention of this Subcommittee that 
on June 19, 2008--transcript available--the relevant 
Subcommittee of Foreign Affairs had a hearing titled ``Genetic 
and Other Human Modification Technologies: Sensible 
International Regulation or a New Kind of Arms Race?'' And in 
fact the analogy to what we're talking about here is the only 
other technology that was equally explosive perhaps, and that 
is nuclear weapons technology. In 1939 Albert Einstein wrote to 
Roosevelt saying what was possible and policymakers had only 
six years before that technology literally exploded onto the 
scene. Thank God we've got a little bit more time but the 
Nonproliferation Treaty took many decades after 1939 or after 
1945 and I think could be a good model for what we need here.
    Dr. Kahn, it may be too long to list but I don't know 
whether America is exactly number one in this technology or 
whether Britain or China might be slightly ahead, but we're all 
within, I think, a few years, but there are a whole bunch of 
other countries either at that level or maybe four or five 
years behind. Can you even list the countries that within five 
years could be where we are now?
    Dr. Kahn. I don't know--
    Mr. Sherman. Are we talking a dozen, two dozen?
    Dr. Kahn. I'm not sure that it's--that I or anybody could 
do that, and in fact, I'm not sure that it even requires 
countries. It's individuals who have access to the capacity--
    Mr. Sherman. Um-hum.
    Dr. Kahn. --which, as Dr. Doudna has said, is actually 
fairly democratic I think was the term that she used. So in a 
way, you know, it's about where people have the laboratory 
capacity and that's almost anywhere in the world.
    Mr. Sherman. Well, thank God nuclear weapons take an 
industrial scale. And although they reflect only 1945 
technology, we've seen in Iran that you have to do something 
big, you have to spend billions of dollars, it has to be 
visible to the world that you're doing something. You seem to 
be saying that what we're concerned about here could be a lot 
cheaper and take place in a laboratory basement?
    Dr. Kahn. And I'll let my colleagues speak to the concrete 
answer to that question but I think exactly that kind of point 
is really important for how we think about what appropriate 
oversight, regulation, guidelines need to be--
    Mr. Sherman. And I want to pick up on Mr. Foster's question 
about time frame, but one of the concerns we will have is that 
countries will see this as, oh my God, it might be terribly 
unethical but it gives us a leg up militarily or economically. 
Damn the torpedoes.
    Leaving aside engineering intelligence and looking to 
things that would--other than that that would affect soldiers, 
soldier is a little better if they've got courage, stamina, and 
strength. We're already at a point where drugs are going to be 
used by various militaries to impart those characteristics to 
their soldiers but then we can go further to genetic 
engineering. What is the time frame before there's genetic 
engineering that would do the simplest of--I don't know which 
of those three is easiest--would give a soldier either more 
strength or more stamina or, say, more courage, more 
willingness to charge out? Is there any way to say that that 
soldier is five years from now, fifteen years from now, or are 
we in the world of science fiction? Anybody venture a guess?
    Dr. McNally. Science fiction.
    Mr. Sherman. Okay. Well, we'll be in a position I think 
next decade where at least--well, already many militaries are 
using drugs on their soldiers and then the next step will be to 
use the next element of medical technology, not drugs, but 
genetic engineering.
    Does anyone have--I mean we're--the first and most ethical 
use of this technology is to remedy maladies. The next step 
will be to allow parents to have kids that have all the best 
characteristics of anyone in their family or in the world. And 
then we'll go to giving kids unprecedented capacities, and at 
that point we're talking maybe human, maybe trans-human. What--
does anyone here have any view as to how long it will be before 
we can affect the intelligence of either people--either adults 
or of germ cell--the germ line?
    Dr. McNally. I'll dive in.
    Mr. Sherman. Dr. McNally is coming close.
    Dr. McNally. I'll dive in.
    As Dr. Doudna has said, the limitations of engineering 
things like intelligence--
    Mr. Sherman. Um-hum.
    Dr. McNally. --are far more limited by our genetic 
observations--
    Mr. Sherman. Um-hum.
    Dr. McNally. --than they are by our capacity to do genome 
engineering. We have actually very large scale genetic projects 
ongoing right now, the 1,000 Genomes Project in the next year 
or two we'll be looking at a million human sequences and a lot 
of information connected to it. And the simple answer is that 
traits like intelligence are not single-gene--
    Mr. Sherman. Yeah.
    Dr. McNally. They're probably not even entirely genetic is 
what people are referring to on a regular basis right now. 
There's a whole series of articles written about the missing 
heritability for many different traits, things that we thought 
were genetic when it turns out we look at the genes, they may 
not be so genetic or they are a very high complexity.
    Mr. Sherman. So the technology--it's interesting because in 
nuclear weapons there's two components. One is weaponizing the 
highly enriched uranium, which turns out to be the easy part. 
It seems the most dangerous. Oh, my God, you're going to turn 
it into a nuclear weapon. The real hard part and the decider as 
to which countries have nuclear weapons technology is the 
ability to enrich uranium.
    Mr. Moolenaar. The gentleman's time is--
    Mr. Sherman. If I--it seems like what you're describing is 
a situation where the roadmap to what genes do--have what 
characteristics is the hard part and the part that will 
determine and the actual snipping and replacing and editing, 
you guys have that down.
    I yield back.
    Mr. Moolenaar. Thank you.
    Ms. Bonamici.
    Ms. Bonamici. Thank you very much, Mr. Chairman. And thank 
you to the Chairman and Ranking Member for holding this hearing 
about this important topic, and I--my absence for most of the 
hearing was only because I was in another hearing. It does not 
indicate my lack of interest in the subject.
    And I'm really glad that we have this, I agree, very 
incredibly qualified panel and I especially appreciate the 
gender diversity. As someone who works on education issues and 
trying to get more women in STEM, thank you for having a 
balanced panel.
    So I missed--a lot of the questions have already been asked 
but there's a couple of things that I wanted to follow up on. 
There's been a lot of attention on using gene editing 
technologies in human embryos, and of course we--with the press 
from what happened in China, that's getting attention. But I 
know that a lot of the research is not in human embryos, so 
could you discuss how the technologies are being used in 
research today in other areas, including in organisms other 
than humans? And also can you talk about the potential promise 
from sectors other than healthcare, energy, for example?
    Dr. Doudna. Okay. Well, I'll--I can take a stab at that. So 
you're absolutely right that the technology is being widely 
employed in many different kinds of cells and organisms, and 
I'll just give you a couple of examples. I think that in plant 
biology this is going to be, you know, equally impactful as the 
kind of thing that--kind of applications that we're talking 
about here in human health in terms of enabling very, you know, 
widespread introduction of genes into plants that could be 
beneficial especially for dealing with climate change and other 
kinds of environmental impacts in plants. And I'm just--I'm not 
a plant biologist but I'm saying this based on conversations 
I've had with people that are already doing those kinds of 
experiments and are extremely excited about the way that that 
research has been enabled.
    And then you mentioned biofuels and I think that's very 
interesting because I'm aware of several groups that are 
actually using this kind of genome engineering for what we call 
systems biology, basically being able to make large-scale 
changes in the genomes of organisms that will be useful for 
producing various kinds of chemicals, including, you know, 
biofuels and other very important materials that can be 
difficult to obtain in other ways.
    Ms. Bonamici. Terrific. And I know Dr. Dzau wants to----
    Dr. Dzau. This in fact is a very important area. Today 
we're talking about human genome editing but I'm glad you 
raised the question about the usage in other living organisms. 
Mr. Sherman asked about, you know, what will be the misuse, if 
you will, but we do have to think about, you know, the use in 
plants, insects, et cetera, you know, what happens if there's 
misuse and what would happen to the environmental impact and 
who actually gets to decide who's going to do what? And there 
are more commercial sources I believe--opportunity for 
commercialization in changing coffee beans or whatever that you 
can imagine.
    So this is an area that in fact--called gene drive that, 
again, National Academies are looking very carefully at this 
and how to regulate this. So I do think this is to me a very 
important area even though our focus is on human gene editing.
    Ms. Bonamici. Terrific. And I'm going to slightly change 
the topic.
    Dr. Doudna--did I say your name properly?
    Dr. Doudna. Yes.
    Ms. Bonamici. I know you cofounded several startup 
companies and we've had conversations in this Committee before 
about the challenges in trying to launch companies but also 
transferring academic research into the marketplace. So can you 
chat a little bit about that and what the challenges have been 
and what the Federal Government can do to help with 
transferring academic research into the marketplace?
    Dr. Doudna. Yeah, this is a very important area and I'm a 
real newbie to it. This is actually the first time that 
research in my lab has led to something that, you know, had 
clear commercial applications. So I can tell you my experience, 
you know, being involved in starting companies and raising 
money for companies. We've had different experiences I would 
say. I've been involved in three different startups around this 
technology so far. And I've had a lot of help with one of them 
in particular through a--what would I call it? I guess it's a 
biomedical research initiative in the Bay area that was 
actually funded in part by the State of California. And what 
that does is to give people like me who know nothing about, you 
know, starting a business some training. We got access to some 
legal advice early on. We--I think this Institute paid for the 
incorporation of the company initially and then gave us some 
support in terms of writing for federal funding for the 
company.
    I think this kind of support is really important. And as I 
talk to my colleagues around the country who are trying to do 
similar things, commercializing work coming out of their 
academic labs, I hear over and over how all of us who are in 
the academic world could really benefit from that kind of level 
of support. I'm not sure if it's something that happens at the 
federal level or if it's better done lower down, hard to say.
    Ms. Bonamici. Thank you so much.
    And my time is expired. I yield back, Mr. Chairman. Thank 
you.
    Mr. Moolenaar. Thank you.
    And now I want to recognize Mr. Palmer.
    Mr. Palmer. A couple of quick questions. Most of this 
discussion I think has been about government research. You're 
talking about convening an international summit. I just want to 
make sure that when we're talking about implementing safeguards 
that this includes the private sector, that they're not running 
out there as mavericks. Is that correct?
    Dr. Dzau. Certainly our intention, if you look carefully at 
the way we put our advisory committee together, we have people 
who have great industry experience on it and our intention is 
to include industry and, you know, people in the commercial 
sector to be in this discussion.
    Mr. Palmer. Thank you. I yield the balance of my time, Mr. 
Chairman.
    Mr. Moolenaar. Thank you.
    I now recognize Mr. Foster.
    Mr. Foster. Thank you.
    It's my understanding that very often there's a computing 
bottleneck in our ability to analyze genomes. And I guess, Dr. 
McNally, can you tell the Committee about some of the work 
you've done at Argonne----
    Dr. McNally. Yeah.
    Mr. Foster. --which is a facility shared by the Ranking 
Member and myself.
    Dr. McNally. Yeah, so right now it's--since more than a 
year-and-a-half, it's been very possible to sequence a human 
genome with the consumable cost being about $1,000, which is an 
amazingly low price for a single genome. That doesn't include 
the cost of analysis, and so the real rate-limiting step right 
now is the time it takes to analyze genomes.
    So what we've done is we've actually, working together with 
Argonne National Labs, the University of Chicago, and now 
Northwestern, we've actually taken a Cray XE6 supercomputer 
that's at Argonne and outfitted it with all the computing code 
that's available through the Broad Institute so that we can 
now, for example, analyze 250 genomes in a weekend if they give 
us the whole machine to work. So that dramatically accelerates 
our ability to screen through three billion bases of an 
individual genome and score it's four million differences that 
exist in each one of those genomes, the vast majority of which 
are rare and private.
    Mr. Foster. And--well, thank you. That's very interesting. 
I guess another application of federal investments that are 
having an unintended benefit.
    I guess for Dr. Kahn, has anyone ever taken the bull by the 
horns and actually attempted to draft legislation or 
international treaties, you know, at regulating human genetic 
engineering or other genetic engineering in the environment?
    Dr. Kahn. That's a good question. I don't know that there 
have been legislation or, you know, bills proposed and I don't 
know about international treaties. There certainly have been 
efforts to craft guidelines but they're nonbinding by entities 
like the World Health Organization or the World Medical 
Association, which has crafted actually fairly well-known 
guidelines on human subjects research which is called 
Declaration of Helsinki, which tends to be followed but in a 
voluntary way and then built into regulation at the national 
level. But I don't think that there has been any successful 
international regulation.
    Mr. Foster. All right, or even proposed--just--you know, 
lists enumeration of all of the issues that you have to resolve 
when you write rules.
    Dr. Kahn. Yeah, I'm not aware, although not a historian of 
science. That's a really interesting question that I--maybe 
I'll do some looking and see if I can get back to you.
    Mr. Foster. I would appreciate that.
    Dr. Kahn. Sure.
    Mr. Foster. And do you understand right now if you're on a 
hospital ship in international waters, who's the regulator?
    Dr. Kahn. That's the right kind of question to be asking. 
And this sort of issue came up when there was a supposed 
cloning of a human being. You may remember back in--when that--
whenever that was, the late '90s, early 2000s--
    Mr. Foster. All the----
    Dr. Kahn. Yeah, which turned out to be a hoax. But one of 
the claims was it was being done in international waters and 
out of the reach of any of the national regulations or 
governance structure, which, you know, in a way was a helpful 
aha moment, right? You need to think about what to do when all 
you need is a well-fitted ship that has the laboratory on board 
and you're outside of the--any restrictions that an 
international treaty or national government might employ.
    Mr. Foster. Okay. And I guess one last question. The issue 
of gene drives has entered the news and interestingly is with 
the potential to sort of take over the genome of an entire 
species in the wild in the course of, you know, a few dozens of 
generations. And so this obviously has huge environmental 
effects and has to be internationally regulated presumably 
because of, you know, insects don't normally, you know, obey 
national borders.
    And so I was just wondering if that is an area where, for 
example, this Committee might interestingly have a separate 
branch of investigation that all of the applications of this 
technology to plants and animals in the wild and in the 
laboratory?
    Yes, Dr. Dzau.
    Dr. Dzau. Mr. Foster, as I mentioned, I think the National 
Academies is undertaking such a study. We'd be happy to send 
you all the materials, and in fact, as we look at what we are 
potentially covering and not covering, we can have a 
conversation about what else should be done.
    Mr. Foster. Okay. And so the charge is complete for the 
study or is it----
    Dr. Dzau. Yes.
    Mr. Foster. --ongoing? All right. I'd appreciate that. 
Thank you.
    I yield back.
    Mr. Moolenaar. Thank you.
    And I'd like to thank our witnesses for the testimony 
today, outstanding, and all the Members for the questions. The 
record will remain open for two weeks for additional comments 
and written questions from Members. The witnesses are excused 
and this hearing is adjourned.
    [Whereupon, at 3:56 p.m., the Subcommittee was adjourned.]

                               Appendix I

                              ----------                              


                   Answers to Post-Hearing Questions




                   Answers to Post-Hearing Questions
Responses by Dr. Elizabeth McNally
[GRAPHIC NOT AVAILABLE IN TIFF FORMAT]


                              Appendix II

                              ----------                              


                   Additional Material for the Record




          Statement submitted by full Committee Ranking Member
                         Eddie Bernice Johnson

    Thank you, Madam Chairwoman for holding this hearing, and I 
want to join you in welcoming our distinguished panel of 
witnesses.
    This afternoon we are talking about new gene editing 
technologies that have promising applications in fields ranging 
from medicine to energy to agriculture. The Chinese research 
paper that prompted this hearing highlights the need to have a 
serious examination of the science, safety, and ethics of gene 
editing technologies.
    I want to thank the Chairwoman and Ranking Member for 
putting together this hearing with an impressive panel of 
expert witnesses. Additionally, I want to acknowledge Dr. 
Foster, whom I understand was instrumental in advocating for 
this hearing.
    The technologies we are discussing today can alter DNA--the 
blueprint of life. There are significant safety, efficacy, and 
ethical issues concerning these technologies. What are the 
ethical uses of these technologies? Is it ethical to use them 
if they could cure a disease? What if they just treated a 
disease?
    Any applications that would alter germline cells, where 
changes are passed down through generations, have additional 
ethical issues. For example, is it ethical for the current 
generation to consent to changes for a future one?
    Although today we are discussing human applications, we 
should not forget that these same technologies also have great 
potential for use in energy and agriculture. Gene editing could 
be used to create biofuels and new crops. Responsible 
applications of these technologies could lead to significant 
economic growth if the U.S. takes the lead in research and 
transferring that research to the private sector.
    I look forward to hearing from the witnesses about the 
state of the gene editing science and potential applications. 
Additionally, the U.S. needs to take a leadership role in 
addressing relevant ethical issues so I am glad that the panel 
includes a bioethicist to help us better understand what is 
involved.
    Finally, I look forward to hearing about the National 
Academies' plans to address these and other important questions 
surrounding this emerging research area.
    Thank you and I yield the balance of my time.

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