Efficacious genome editing in infant mice with glycogen storage disease type Ia
在患有 Ia 型糖原贮积症的幼鼠中进行有效的基因组编辑
期刊:JCI Insight
影响因子:6.1
doi:10.1172/jci.insight.181760
Arnson, Benjamin; Ilich, Ekaterina; von Beck, Troy; Li, Songtao; Brooks, Elizabeth D; Gheorghiu, Dorothy; He, Gordon; Weinrub, Matthew; Chan, Sze Ying; Kang, Hye-Ri; Courtney, David; Everitt, Jeffrey I; Cullen, Bryan R; Koeberl, Dwight D