AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (ribosome profiling)
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https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE179274
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资源简介：
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). Liver ribosome profilings in sup-tRNA treated and untreated homozygous Idua-W401X mice and patient fibroblasts.
创建时间：
2022-04-22
