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

Author:

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

Funder

Genethon

French Muscular Dystrophy Association

European Union’s research and innovation program

ASTRE laboratories of the Essonne

Italian Telethon

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference54 articles.

1. Glycogen Storage Disease Type III;Dagli,2010

2. Glycogen storage disease type III: diagnosis, genotype, management, clinical course and outcome;Sentner;J. Inherit. Metab. Dis.,2016

3. Metabolic disorders affecting muscle;Di Mauro,2004

4. Clinical, biochemical and genetic features of glycogen debranching enzyme deficiency;Lucchiari;Acta Myol.,2007

5. Cornstarch therapy in a patient with type III glycogen storage disease;Borowitz;J. Pediatr. Gastroenterol. Nutr.,1987

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