Molecular Therapy - Nucleic Acids
 2024
DOI: 10.1016/j.omtn.2024.102220
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Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells

Chloe L. Christensen,
Shih-Hsin Kan,
Perla Andrade-Heckman
et al.
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