Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Huntington's disease (HD)

Brain-derived neurotrophic factor (BDNF); noggin (NOG)

Rodent and primate studies suggest overexpression of BDNF and NOG could help treat HD. In a mouse model for HD, intracerebroventricular injection of an adeno-associated virus (AAV) vector to induce BDNF and NOG overexpression resulted in recruitment of new medium spiny neurons that matured and functionally integrated with existing neurons. In the model, BDNF and NOG overexpression delayed disease progression and increased overall survival compared with AAV-mediated overexpression of a control protein. In normal nonhuman primates, the AAV vector also generated new neurons. Next steps could include developing a therapeutic strategy to upregulate BDNF and NOG in the CNS.

SciBX 6(26); doi:10.1038/scibx.2013.664
Published online July 11, 2013

Patent and licensing status unavailable

Benraiss, A. et al. Cell Stem Cell; published online June 6, 2013;
doi:10.1016/j.stem.2013.04.014
Contact: Steven A. Goldman, University of Rochester Medical Center, Rochester, N.Y.
e-mail:
steven_goldman@urmc.rochester.edu

Contact: Abdellatif Benraiss, same affiliation as above
e-mail:
abdellatif_benraiss@urmc.rochester.edu