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Glial Cell Line-Derived Neurotrophic Factor Opposite Strand (GDNFOS) for Treatment of Neurodegenerative Diseases

Posted Jul 26 2012 8:00pm

Description of Invention:
Glial cell line-derived neurotrophic factor (GDNF) is a small human protein encoded by the GDNF gene. GDNF has been effective therapy in laboratory animal models of Parkinson's disease and protects several types of neurons in the brain and peripheral nervous system. The NIDA inventors have discovered primate-specific GDNFOS, encoded by the opposite strand of glial cell derived neurotrophic factor (GDNF) gene. The GDNFOS gene encodes for novel peptides that was found to be reduced in human middle temporal gyrus of Alzheimer's disease brains. These secreted growth proteins have potential neurotrophic activity and they might play a synergistic role in neuroprotective effects of GDNF in human brain. The NIDA inventors have also developed antibody against GDNFOS3 and generated compounds that have potential pharmaceutical use. The compounds consist of GDNFOS nucleic acid transcripts, GDNFOS protein or a functional fragment for treatment of human neurodegenerative diseases.

  • Synergistic role in neuroprotective effects of GDNF
  • Alzheimer's disease, Parkinson's disease, Amyotrophic lateral sclerosis, multiple sclerosis and diseases of peripheral organs such as diabetes mellitus type 1

  • Secreted novel growth peptides
  • An antibody against GDNFOS3 was developed

Development Status:
  • Early-stage
  • Pre-clinical
  • In vitro data available

Qing Rong Liu (NIDA)
Mikko T Airavaara (NIDA)
Barry J Hoffer (NIDA)
Brandon K Harvey (NIDA)

Patent Status:
HHS, Reference No. E-044-2012/0
US, Application No. 61/619,296 filed 02 Apr 2012

Relevant Publication:
  1. Airavaara M, et al. [ PMID 22081608 ]

Collaborative Research Opportunity:
The National Institute on Drug Abuse is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize GDNFOS peptide and non-coding RNAs as therapeutic agents for neurodegenerative diseases. For collaboration opportunities, please contact Vio Conley at .

For Licensing Information Please Contact:
Betty Tong Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-594-6565
Fax: 301-402-0220

Ref No: 2465

Updated: 07/2012

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