Combined overexpression of ATXN1L and mutant ATXN1 knockdown by AAV rescue motor phenotypes and gene signatures in SCA1 mice

Combined overexpression of ATXN1L and mutant ATXN1 knockdown by AAV rescue motor phenotypes and gene signatures in SCA1 mice

Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disease caused by a (CAG) repeat expansion in the coding sequence of ATXN1. The primary mechanism of disease in SCA1 is toxic gain of function by polyglutamine-expanded mutant ATXN1 and is compounded by partial loss of w...

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Bibliographic Details
Main Authors: Ellie M. Carrell, Megan S. Keiser, Ashley B. Robbins, Beverly L. Davidson
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
spinocerebellar ataxia type 1
ataxin-1
ataxin-1-like
AAV
miRNA
RNAi
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000523

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http://www.sciencedirect.com/science/article/pii/S2329050122000523

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