Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
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Mol Ther. 2024 Sep 17:S1525-0016(24)00606-3. doi: 10.1016/j.ymthe.2024.09.024. Online ahead of print.
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PMID: 39295144