Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells
Infantile-onset Pompe disease (IOPD) results from pathogenic variants in the GAA gene, which encodes acid α-glucosidase. The correction of pathogenic variants through genome editing may be a valuable one-time therapy for PD and improve upon the current standard of care. We performed adenine base edi...
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| Main Authors: | , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2024-06-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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