Reduction of mutant ATXN1 rescues premature death in a conditional SCA1 mouse model

Spinocerebellar ataxia type 1 (SCA1) is an adult-onset neurodegenerative disorder. As disease progresses, motor neurons are affected, and their dysfunction contributes toward the inability to maintain proper respiratory function, a major driving force for premature death in SCA1. To investigate the...

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Bibliographic Details
Main Authors: Ciaburri, N.A (Author), Nitschke, L. (Author), Orengo, J.P (Author), Orr, H.T (Author), van der Heijden, M.E (Author), Zoghbi, H.Y (Author)
Format: Article
Language:English
Published: American Society for Clinical Investigation 2022
Online Access:View Fulltext in Publisher