Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease

Title
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
Authors
Keywords
-
Journal
Human Gene Therapy Clinical Development
Volume 25, Issue 3, Pages 134-163
Publisher
Mary Ann Liebert Inc
Online
2014-09-20
DOI
10.1089/humc.2014.2514

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