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: Carrell, E.M (Author), Davidson, B.L (Author), Keiser, M.S (Author), Robbins, A.B (Author)
Format: Article
Language:English
Published: Cell Press 2022
Subjects:
AAV
Online Access:View Fulltext in Publisher