[Federal Register Volume 69, Number 243 (Monday, December 20, 2004)]
[Notices]
[Pages 75992-75993]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 04-27782]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Government-Owned Inventions; Availability for Licensing

AGENCY: National Institutes of Health, Public Health Service, DHHS.

ACTION: Notice.

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SUMMARY: The inventions listed below are owned by an agency of the U.S. 
Government and are available for licensing in the U.S. in accordance 
with 35 U.S.C. 207 to achieve expeditious commercialization of results 
of federally-funded research and development. Foreign patent 
applications are filed on selected inventions to extend market coverage 
for companies and may also be available for licensing.

ADDRESSES: Licensing information and copies of the U.S. patent 
applications listed below may be obtained by writing to the indicated 
licensing contact at the Office of Technology Transfer, National 
Institutes of Health, 6011 Executive Boulevard, Suite 325, Rockville, 
Maryland 20852-3804; telephone: (301) 496-7057; fax: (301) 402-0220. A 
signed Confidential Disclosure Agreement will be required to receive 
copies of the patent applications.

AAV4 Vector and Uses Thereof

    John A. Chiorini (NHLBI/NIDCR), Robert M. Kotin (NHLBI), Brian 
Safer (NHLBI). U.S. Patent 6,468,524 issued 22 Oct 2002 (DHHS Reference 
No. E-071-2000/0-US-01).
    Licensing Contact: Jesse Kindra; (301) 435-5559; 
[email protected].
    The invention described and claimed in this patent application 
relates to the delivery of heterologous nucleic acids or genes to 
particular target cells. In particular, the application relates to 
methods of delivering a heterologous nucleic acid or gene of interest 
to particular target cells using Adeno-Associated Virus of serotype 4 
(AAV4). The particular target cells identified are the ependymal cells 
of the brain. The methods described herein may be useful in carrying 
out gene therapy for diseases of the brain or central nervous system.
    This work has been published in part at Davidson, BL, et al. 
``Recombinant adeno-associated virus type 2, 4, and 5 vectors: 
transduction of variant cell types and regions in the mammalian central 
nervous system'' PNAS USA 97(7):3428-32 (March 28, 2000).
    In addition, PHS owns additional intellectual property related to 
this technology describing an AAV4-based vector system. The material 
contained in the patent application has been published as WO 98/11244 
(March 19, 1998) and the research corresponding thereto has been 
published in J. Virology 71(9): 6823-33 (Sept 1997).

AAV5 Vector for Transducing Brain Cells and Lung Cells

    John A. Chiorini (NHLBI/NIDCR), Robert M. Kotin (NHLBI).
    U.S. Patent Application No. 09/533,427 filed 22 Mar 2000 (DHHS 
Reference No. E-072-2000/0-US-01).
    Licensing Contact: Jesse Kindra; (301) 435-5559; 
[email protected].
    The invention described and claimed in this patent application is 
related to the delivery of heterologous nucleic acids or genes to 
particular target cells. In particular, the application relates to 
methods of delivering a heterologous nucleic acid or gene of interest 
to particular target cells using an Adeno-Associated Virus of serotype 
5 (AAV5). The particular target cells identified include the alveolar 
cells of the lung and cerebellar and ependymal cells of the brain. The 
methods described herein may be useful in carrying out gene therapy 
related to diseases of the brain or central nervous system and the 
respiratory tract.
    This work has been published, in part, at Davidson BL, et al. PNAS, 
USA 97(7):3428-32 (March 28, 2000) and Zabner J, et al. J Virol. 
74(8):3852-8 (April 2000).
    In addition to this patent application, PHS owns additional 
intellectual property related to this technology. The patent 
application has been published as WO 99/61601 on December 2, 1999 and 
the research corresponding thereto has been published at Chiorini JA, 
et al. J. Virol. 73(5): 4293-98 (May 1999) and Chiorini JA, et al. J. 
Virol. 73(2): 1309-19 (Feb. 1999).

TTP as a Regulator of GM-CSF mRNA Deadenylation and Stability

    Ester Carballo-Jane, Wi S. Lai, Perry J. Blackshear (NIEHS). U.S. 
Provisional Application No. 60/148,810 filed 13 Aug 1999 (DHHS 
Reference No. E-204-1999/0-US-01); PCT Application No. PCT/US00/22199 
filed 12 Aug 2000, which published as WO 01/12213 on 22 Feb 2001 (DHHS 
Reference No. E-204-1999/0-PCT-02); U.S. Patent Application No. 10/
049,586 filed 12 Feb 2002 (DHHS Reference No. E-204-1999/0-US-03).
    Licensing Contact: Jesse Kindra; (301) 435-5559; 
[email protected].
    The disclosed invention provides materials and methods to treat 
granulocytopenia (low white cell count in the blood) which is 
characterized by a reduced number of granulocytes (relative) or an 
absence of granulocytes (absolute). This condition is commonly 
associated with cancer chemotherapy, but is seen less frequently in a 
number of conditions including the use of propylthiouracil, 
radiotherapy for marrow ablation for bone marrow transplantation, 
aplastic anemia, systemic lupus erythematosus, AIDS and a variety of 
other situations. The

[[Page 75993]]

invention proposes a method to increase GM-CSF levels in a treated 
subject, and this increase is achieved by inhibiting the degradation of 
GM-CSF messenger RNA (mRNA). Tristetraprolin (TTP) is one member of a 
family of cys-cys-cys-his (CCCH) zinc finger proteins, and it is a 
factor that binds to and causes the instability of GM-CSF mRNA. Methods 
are provided for the development of screening assays for molecules that 
inhibit the binding of TTP and its related proteins to GM-CSF mRNA, or 
otherwise inhibit the effect of TTP to promote breakdown of the mRNA, 
leading in turn to increased mRNA stability and enhanced production of 
GM-CSF. Compounds identified by such screens, and their derivatives, 
could be useful in treating granulocytopenia from whatever cause.
    Additional information about this technology may be found in the 
following research articles:
    Carballo, E, Lai, WS and Blackshear, PJ. Evidence that 
tristetraprolin (TTP) is a physiological regulator of granulocyte-
macrophage colony-stimulating factor (GM-CSF) mRNA deadenylation and 
stability. 2000; Blood 95:1891-1899.
    Lai, WS, Carballo, E, Thorn, JM, Kennington, EA and Blackshear, PJ. 
Interactions of CCCH zinc finger proteins with mRNA. 1. Binding of 
tristetraprolin-related zinc finger proteins to AU-rich elements and 
destabilization of mRNA. 2000; J. Biol. Chem., 275:17827-19837.
    In addition to licensing, the technology is available for further 
development through collaborative research with the inventors via a 
Cooperative Research and Development Agreement (CRADA).

    Dated: December 13, 2004.
Steven M. Ferguson,
Director, Division of Technology Development and Transfer, Office of 
Technology Transfer, National Institutes of Health.
[FR Doc. 04-27782 Filed 12-17-04; 8:45 am]
BILLING CODE 4140-01-P