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

ABSTRACT: ATXN2 has emerged as an exciting therapeutic target for neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia type 2 (SCA2), as lowering its levels via genomic knockout or anti-sense oligonucleotide (ASO) treatment has been shown to mitigate disease phenotypes and led to a current clinical trial of ATXN2 ASOs for treatment of ALS in humans. To identify additional ways to lower ataxin‑2 protein levels, we performed a genome-wide fluorescence activated cell sorting (FACS)-based CRISPR screen in human cells and identified multiple components of the lysosomal vacuolar ATPase (v‑ATPase) as modifiers of ataxin‑2 levels. This dataset contains the RNA-sequencing data and CRISPR screen data used to support the conclusions from this study.

ORGANISM(S): Homo sapiens

PROVIDER: GSE189417 | GEO | 2022/10/18

REPOSITORIES: GEO

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Targeting RTN4/NoGo-Receptor reduces levels of ALS protein ataxin-2

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