Article
A fully humanized transgenic mouse model of Huntington disease.
Human molecular genetics - 1 Jan 2013
Southwell Amber L, Warby Simon C, Carroll Jeffrey B, Doty Crystal N, Skotte Niels H, Zhang Weining, Villanueva Erika B, Kovalik Vlad, Xie Yuanyun, Pouladi Mahmoud A, Collins Jennifer A, Yang X William, Franciosi Sonia, Hayden Michael R
Abstract excerpt
Silencing the mutant huntingtin gene (muHTT) is a direct and simple therapeutic strategy for the treatment of Huntington disease (HD) in principle. However, targeting the HD mutation presents challenges because it is an expansion of a common genetic element (a CAG tract) that is found throughout the genome. Moreover, the HTT protein is important for neuronal health throughout life, and silencing strategies that...
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