AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (tRNA_charging)
收藏NIAID Data Ecosystem2026-03-13 收录
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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). targeted tRNA amplicon sequencing of sup-tRNA treated and untreated homozygous Idua-W401X mice
创建时间：
2022-04-22
