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###### Department of Health and Human Services

###### National Institutes of Health 

# AGENCY:

National Institutes of Health, Public Health Service, DHHS. 

# ACTION:

Notice. 

# 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;                     *kindraj@mail.nih.gov*.                 

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;                     *kindraj@mail.nih.gov*.                 

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;                     *kindraj@mail.nih.gov*.                 

                    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                           ( print 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