Ontology highlight
ABSTRACT:
SUBMITTER: Kim G
PROVIDER: S-EPMC9664452 | biostudies-literature | 2022 Oct
REPOSITORIES: biostudies-literature
Cell reports 20221001 4
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-2 levels could lead to cheaper or less invasive therapies and elucidate how ataxin-2 is normally regulated. Here, we perform a genome-wide fluorescence-activated cell sorting (FACS)-based CRISPR-Cas9 ...[more]