Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells
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Elsevier BV
Reference52 articles.
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Cited by 3 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Precision medicine in action for Pompe disease;Molecular Therapy - Nucleic Acids;2024-09
2. Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells;Molecular Genetics and Metabolism;2024-09
3. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy;Molecular Diagnosis & Therapy;2024-08-12
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