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RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1

OBJECTIVE: Spinocerebellar ataxia type 1 is an autosomal dominant fatal neurodegenerative disease caused by a polyglutamine expansion in the coding region of ATXN1. We showed previously that partial suppression of mutant ataxin‐1 (ATXN1) expression, using virally expressed RNAi triggers, could preve...

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Bibliografiske detaljer
Udgivet i:Ann Neurol
Main Authors: Keiser, Megan S., Monteys, Alejandro Mas, Corbau, Romuald, Gonzalez‐Alegre, Pedro, Davidson, Beverly L.
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2016
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5115960/
https://ncbi.nlm.nih.gov/pubmed/27686464
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24789
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