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Gene therapy for glycogen storage diseases

The focus of this review is the development of gene therapy for glycogen storage diseases (GSDs). GSD results from the deficiency of specific enzymes involved in the storage and retrieval of glucose in the body. Broadly, GSDs can be divided into types that affect liver or muscle or both tissues. For...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Kishnani, Priya S, Sun, Baodong, Koeberl, Dwight D
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6796997/
https://ncbi.nlm.nih.gov/pubmed/31227835
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz133
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