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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

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Benraiss, A. et al. Cell Stem Cell; published online June 6, 2013;
Contact: Steven A. Goldman, University of Rochester Medical Center, Rochester, N.Y.

Contact: Abdellatif Benraiss, same affiliation as above