Astrocyte transduction is required for rescue of behavioral phenotypes in the YAC128 mouse model with AAV-RNAi mediated HTT lowering therapeutics

Huntington's disease (HD) is a fatal autosomal dominant neurodegenerative disease caused by a CAG expansion, which translates into an elongated polyglutamine (polyQ) repeat near the amino-terminus of the huntingtin protein (HTT). This results in production of a toxic mutant huntingtin protein (...

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Bibliographic Details
Main Authors: Lisa M. Stanek, Jie Bu, Lamya S. Shihabuddin
Format: Article
Language:English
Published: Elsevier 2019-09-01
Series:Neurobiology of Disease
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996119301044