Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
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Mol Ther. 2024 Nov 6;32(11):3847-3864. doi: 10.1016/j.ymthe.2024.09.024. Epub 2024 Sep 17.
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PMID: 39295144