Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease

Published 2019 View Full Article

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Title
Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease
Authors
Keywords
Pompe disease, acid alpha-glucosidase deficiency, gene therapy, AAV-PHP.B vector, glycogen storage, CNS, neurological deficits
Journal
Molecular Therapy-Methods & Clinical Development
Volume 12, Issue -, Pages 233-245
Publisher
Elsevier BV
Online
2019-01-27
DOI
10.1016/j.omtm.2019.01.006

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