Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

doi:10.1016/j.ymthe.2017.12.019

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Article

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Molecular therapy : the journal of the American Society of Gene Therapy - 7 Mar 2018

Vidal Patrice, Pagliarani Serena, Colella Pasqualina, Costa Verdera Helena, Jauze Louisa, Gjorgjieva Monika, Puzzo Francesco, Marmier Solenne, Collaud Fanny, Simon Sola Marcelo, Charles Severine, Lucchiari Sabrina, van Wittenberghe Laetitia, Vignaud Alban, Gjata Bernard, Richard Isabelle, Laforet Pascal, Malfatti Edoardo, Mithieux Gilles, Rajas Fabienne, Comi Giacomo Pietro, Ronzitti Giuseppe, Mingozzi Federico

Abstract excerpt

Glycogen storage disease type III (GSDIII) is an autosomal recessive disorder caused by a deficiency of glycogen-debranching enzyme (GDE), which results in profound liver metabolism impairment and muscle weakness. To date, no cure is available for GSDIII and current treatments are mostly based on diet. Here we describe the development of a mouse model of GSDIII, which faithfully recapitulates the main features of...

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PMID: 29396266
DOI: 10.1016/j.ymthe.2017.12.019
PMCID: PMC5910667

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