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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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Bibliografiske detaljer
Udgivet i:Hum Mol Genet
Main Authors: Kishnani, Priya S, Sun, Baodong, Koeberl, Dwight D
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2019
Fag:
Online adgang: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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