Article
Nonallele specific silencing of ataxin-7 improves disease phenotypes in a mouse model of SCA7.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2014
Ramachandran Pavitra S, Boudreau Ryan L, Schaefer Kellie A, La Spada Albert R, Davidson Beverly L
Abstract excerpt
Spinocerebellar ataxia type 7 (SCA7) is a late-onset neurodegenerative disease characterized by ataxia and vision loss with no effective treatments in the clinic. The most striking feature is the degeneration of Purkinje neurons of the cerebellum caused by the presence of polyglutamine-expanded ataxin-7. Ataxin-7 is part of a transcriptional complex, and, in the setting of mutant ataxin-7, there is misregulation...
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