Glycogen Reduction in Myotubes of Late-Onset Pompe Disease Patients Using Antisense Technology

Published 2017 View Full Article

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Title
Glycogen Reduction in Myotubes of Late-Onset Pompe Disease Patients Using Antisense Technology
Authors
Keywords
splicing, antisense, Pompe disease, glycogen storage disease type II, GAA, gene, morpholino
Journal
MOLECULAR THERAPY
Volume 25, Issue 9, Pages 2117-2128
Publisher
Elsevier BV
Online
2017-06-17
DOI
10.1016/j.ymthe.2017.05.019

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