E7-1379. Government-Owned Inventions; Availability for Licensing

AGENCY:

National Institutes of Health, Public Health Service, HHS.

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: [phone]; fax: [phone]. A signed Confidential Disclosure Agreement will be required to receive copies of the patent applications.

Diagnostics and Therapeutics for Hydrocephalus

Description of Technology: Congenital hydrocephalus is a significant public health problem, affecting approximately one in 500 live births in the United States. Congenital hydrocephalus has an adverse effect on the developing brain and may persist as neurological defects in children and adults. Some of these defects may manifest as mental retardation, cerebral palsy, epilepsy and visual disabilities. Improved diagnostics are needed for assessing the risks of developing this debilitating disease.

The inventors have shown that RFX4_v3, a splice variant of the Regulatory Factor X4 (RFX4) transcription factor, is associated with the development of neurological structures. The reduction or absence of RFX4-v3 promotes the development of congenital hydrocephalus. This invention describes RFX4_v3 polypeptides and nucleic acids, as well as methods for detection of RFX4_v3 polymorphisms associated with congenital hydrocephalus. Also described are treatment methods including the RFX4-v3 polypeptide and RFX4-v3 transgenic animals and antibodies.

Applications: Prenatal diagnostic assay for identifying children at risk for congenital hydrocephalus; Genotyping assay for congenital hydrocephalus.

Market: In the United States, the health care costs for congenital hydrocephalus are estimated at $100 million per year.

Development Status: In vitro data are available.

Inventors: Perry J. Blackshear, Darryl C. Zeldin, Joan P. Graves, and Deborah J. Stumpo (NIEHS).

Publications:

1. Perry J. Blackshear et al. Graded phenotypic response to partial and complete deficiency of a brain-specific transcript variant of the winged helix transcription factor RFX4. Development. 2003 Oct;130(19):4539-4552.

2. Donghui Zhang et al. Identification of potential target genes for RFX4_v3, a transcription factor critical for brain development. J Neurochem. 2006 Aug;98(3):860-875.

3. Donghui Zhang et al. Regulatory factor X4 variant 3 (RFX4_v3): a transcription factor involved in brain development and disease. Submitted for publication, Journal of Neuroscience Research.

Patent Status: PCT Application No. PCT/US03/12348 filed 18 Apr 2003, which published as WO 03/088919 on 30 Oct 2003 (HHS Reference No. E-163-2002/2-PCT-01); U.S. Patent Application No. 10/511,362 filed 15 Oct 2004, which published as U.S. 2005/0181369 on 18 Aug 2005 (HHS Reference No. E-163-2002/2-US-02).

Licensing Status: Available for exclusive or nonexclusive licensing.

Licensing Contact: Tara Kirby, Ph.D.; 301/435-4426; [e-mail].

Epithelial Cell Line Expressing a Cystic Fibrosis Phenotype

Description of Technology: Cystic fibrosis (CF) is a common genetic disease that affects the entire body, producing thick, sticky mucus that clogs the lungs, pancreas, and other organs. It is the most common fatal genetic disease in the United States, and is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR).

Researchers at NIEHS have developed a cell line, CF/T43, which was produced by infection of airway epithelial cells isolated from CF patients with an SV40T retrovirus. CF/T43 cells maintain the abnormal ion transport characteristics of CF while having proliferation capability beyond that of a primary epithelial cell culture. Key features of the CF/T43 cell line include the formation of functional tight junctions, reduced apical membrane chloride conductance, and activation of apical chloride channels by calcium ionophores but not by cAMP-dependent agonists. This cell line may be used for elucidation of the mechanisms of CF, testing candidate complementary genes for correction of the observed CF abnormalities, and for developing and testing therapeutic CF drugs.

Applications: Research tool for developing new therapies to treat cystic fibrosis; Research tool for studying the mechanisms of cystic fibrosis.

Inventors: Anton M. Jetten (NIEHS).

Publication: AM Jetten, JR Yankaskas, MJ Stutts, NJ Willumsen, and RC Boucher. Persistence of abnormal chloride conductance regulation in SV40 T transformed cystic fibrosis airway epithelia. Science 1989 Jun 23;244(4911):1472-1475.

Patent Status: U.S. Patent Application No. 07/368,725 filed 21 June 1989, which issued as U.S. Patent No. 5,420,033 on 30 May 1995 (HHS Reference No. E-201-1989/0-US-01).

Licensing Status: Available for exclusive or nonexclusive licensing.

Licensing Contact: Tara Kirby, Ph.D.; 301/435-4426; [e-mail].

Dated: January 20, 2007.

Steven M. Ferguson,

Director, Division of Technology Development and Transfer, Office of Technology Transfer, National Institutes of Health.