Ontology highlight
ABSTRACT:
SUBMITTER: Premasiri AS
PROVIDER: S-EPMC7508178 | biostudies-literature | 2020
REPOSITORIES: biostudies-literature
Premasiri Alan S AS Gill Anna L AL Vieira Fernando G FG
Frontiers in pharmacology 20200908
Repeat expansion mutations in the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Repeat-associated non-AUG translation of this expansion produces dipeptide repeat proteins (DRPs). The arginine containing DRPs, polyGR and polyPR, are consistently reported to be the most toxic. Here we demonstrated that small molecule inhibition of type I protein arginine methyltransferases (PRMT) protects against polyGR and polyPR toxicity. ...[more]