Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2Gangliosidosis in Sandhoff Mice

Published 2016 View Full Article

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Title
Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2Gangliosidosis in Sandhoff Mice
Authors
Keywords
-
Journal
HUMAN GENE THERAPY
Volume 27, Issue 7, Pages 497-508
Publisher
Mary Ann Liebert Inc
Online
2016-05-20
DOI
10.1089/hum.2016.015

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