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

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.

Potential Commercial Applications: Competitive Advantages:
  • 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 Stage:
  • Early-stage
  • Pre-clinical
  • In vitro data available


Qing Rong Liu (NIDA)  ➽ more inventions...

Intellectual Property:
U.S. Pat: 8,999,927 issued 2015-04-07
US Application No. 13/855,533

Airavaara M, et al. PMID 22081608

Collaboration 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 William Dee at

Licensing Contact:
Martha Lubet, Ph.D.
Phone: 240-276-5508

OTT Reference No: E-044-2012/0
Updated: Mar 8, 2017