miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3

Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disorder caused by the expansion of a CAG repeat in the ATXN3 gene. This mutation leads to a toxic gain of function of the ataxin-3 protein, resulting in neuronal dysfunction and atrophy of specific brain regions over time. As ataxin-3 is a...

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Detalles Bibliográficos
Autores principales: Nobre, Rui Jorge, Lobo, Diana D., Henriques, Carina, Duarte, Sonia P., Lopes, Sara M., Silva, Ana C., Lopes, Miguel M., Mariet, Fanny, Schwarz, Lukas K., Baatje, M.S., Ferreira, Valerie, Vallès, Astrid, Pereira de Almeida, Luis, Evers, Melvin M., Toonen, Lodewijk J.A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Original Papers
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9221165/
https://www.ncbi.nlm.nih.gov/pubmed/34878314
http://dx.doi.org/10.1089/nat.2021.0020

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