Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
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Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375.
Sci Transl Med. 2017.
PMID: 29187643
Free PMC article.