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...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Cell Press
2022
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| Subjects: | |
| Online Access: | View Fulltext in Publisher |