AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (mRNA-seq)
收藏NIAID Data Ecosystem2026-03-13 收录
下载链接：
https://www.ncbi.nlm.nih.gov/sra/SRP346802
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: mRNA profiles of sup-tRNA treated and untreated in wildtype mice liver and human Idua+/- fibroblast
创建时间：
2022-04-23
