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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Detalles Bibliográficos
Autores principales: Carrell, Ellie M., Keiser, Megan S., Robbins, Ashley B., Davidson, Beverly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9062622/
https://www.ncbi.nlm.nih.gov/pubmed/35573049
http://dx.doi.org/10.1016/j.omtm.2022.04.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9062622/
https://www.ncbi.nlm.nih.gov/pubmed/35573049
http://dx.doi.org/10.1016/j.omtm.2022.04.004

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