AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (amplicon)

ABSTRACT: In this study, we utilized a mouse model of mucopolysaccharidosis type I (MPS-I) carrying a homozygous nonsense mutation in the Idua gene (Idua-W401X, TGG→TAG) to develop rAAV-delivered sup-tRNA therapeutics that can overcome a pathogenic UAG premature termination codon (PTC).

ORGANISM(S): Mus musculus

PROVIDER: GSE179270 | GEO | 2021/11/30

REPOSITORIES: GEO