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

Autor: Rui Jorge Nobre, Diana D. Lobo, Carina Henriques, Sonia P. Duarte, Sara M. Lopes, Ana C. Silva, Miguel M. Lopes, Fanny Mariet, Lukas K. Schwarz, M.S. Baatje, Valerie Ferreira, Astrid Vallès, Luis Pereira de Almeida, Melvin M. Evers, Lodewijk J.A. Toonen
Rok vydání: 2022
Předmět:
Zdroj: Nucleic Acid Therapeutics. 32:194-205
ISSN: 2159-3345
2159-3337
Popis: Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disorder caused by the expansion of a CAG repeat in the
Databáze: OpenAIRE