Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells

Chloe Christensen, Shih-hsin Kan, Perla Andrade-Heckman, Allisandra K. Rha, Jerry F. Harb, Raymond Y. Wang
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碱基编辑可挽救小儿庞贝病患者衍生细胞中的酸α-葡萄糖苷酶功能
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