Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia

Published 2020 View Full Article

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Title
Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia
Authors
Keywords
Recombinant adeno-associated virus vector, Glucose-6-phosphatase-α variant, Clinical translation, Autophagy impairment
Journal
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
Volume 527, Issue 3, Pages 824-830
Publisher
Elsevier BV
Online
2020-05-17
DOI
10.1016/j.bbrc.2020.04.124

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