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Genome-wide CRISPR screen reveals v-ATPase as a drug target to lower levels of ALS protein ataxin-2

Mutations in the ataxin-2 gene (ATXN2) cause the neurodegenerative disorders amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia type 2 (SCA2). A therapeutic strategy using antisense oligonucleotides targeting ATXN2 has entered clinical trial in humans. Additional ways to decrease ataxin-...

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Detalles Bibliográficos
Autores principales:	Kim, Garam, Nakayama, Lisa, Blum, Jacob A., Akiyama, Tetsuya, Boeynaems, Steven, Chakraborty, Meenakshi, Couthouis, Julien, Tassoni-Tsuchida, Eduardo, Rodriguez, Caitlin M., Bassik, Michael C., Gitler, Aaron D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9664452/ https://www.ncbi.nlm.nih.gov/pubmed/36288714 http://dx.doi.org/10.1016/j.celrep.2022.111508

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