Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

Title
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
Authors
Keywords
-
Journal
Human Gene Therapy Clinical Development
Volume 28, Issue 4, Pages 208-218
Publisher
Mary Ann Liebert Inc
Online
2017-11-21
DOI
10.1089/humc.2017.146

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