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

Internet

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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