Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

Title
Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease
Authors
Keywords
-
Journal
HUMAN GENE THERAPY
Volume 27, Issue 7, Pages 509-521
Publisher
Mary Ann Liebert Inc
Online
2016-05-20
DOI
10.1089/hum.2016.013

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