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Phase I study of liver depot gene therapy in late-onset Pompe disease.
Mol Ther. 2023 Jul 5;31(7):1994-2004. doi: 10.1016/j.ymthe.2023.02.014. Epub 2023 Feb 18.
Mol Ther. 2023.
PMID: 36805083
Clinical Trial.
Immunomodulatory, liver depot gene therapy for Pompe disease.
Bond JE, Kishnani PS, Koeberl DD.
Bond JE, et al.
Cell Immunol. 2019 Aug;342:103737. doi: 10.1016/j.cellimm.2017.12.011. Epub 2017 Dec 29.
Cell Immunol. 2019.
PMID: 29295737
Free PMC article.
Review.
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