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Stimulation of Endogenous Neural Progenitor Cells Slows the Progression of Huntington’s Disease

Huntington’s disease (HD) is a neurodegenerative disorder characterized by the progressive loss of striatal medium spiny projection neurons (MSNs). Abdellatif Benraiss, PhD, and Steven A. Goldman, MD, PhD, at the Department of Neurology of the University of Rochester have just reported in the journal Cell Stem Cell that viral co-expression of two proteins, BDNF and Noggin, strongly enhanced neurogenesis and delayed HD progression. They found the intraventricular delivery of the adeno-associated viruses AAV4-BDNF and AAV4-noggin induced the sustained recruitment of endogenous neural progenitor cells in R6/2 huntingtin mutant mice, a model for HD. The newly generated neurons developed as MSNs that matured and achieved circuit integration, which subsequently slowed deterioration of motor function and prolonged the survival of these HD mice. Importantly, similar findings were also observed in squirrel monkeys given injections of AAV4-BDNF/noggin. Their study thus establishes the feasibility and efficacy of  mobilizing endogenous neural progenitor cells as a strategy for treating HD. This work was supported in part by a NYSTEM IIRP award to Dr. Benraiss (C026425).

Abdellatif Benraiss, Michael J. Toner, Qiwu Xu, Elodie Bruel-Jungerman, Eloise H. Rogers,Fushun Wang, Aris N. Economides, Beverly L. Davidson, Ryoichiro Kageyama, Maiken Nedergaard, and Steven A. Goldman. Sustained Mobilization of Endogenous Neural Progenitors Delays Disease Progression in a Transgenic Model of Huntington’s Disease. Cell Stem Cell 12, 787-799, 2013 June 6.