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

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 di...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5910667/
https://ncbi.nlm.nih.gov/pubmed/29396266
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.12.019
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