AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (amplicon). AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (amplicon)
收藏NIAID Data Ecosystem2026-03-12 收录
下载链接：
https://www.ncbi.nlm.nih.gov/bioproject/PRJNA743041
下载链接
链接失效反馈官方服务：
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). Overall design: Idua amplicon sequencing of sup-tRNA treatedand untreated heterozygous Idua-W401X mice
创建时间：
2021-07-01
