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Gene therapy for glycogen storage diseases

8 Jul 2023

Abstract excerpt

Glycogen storage disorders (GSDs) are inherited disorders of metabolism resulting from the deficiency of individual enzymes involved in the synthesis, transport, and degradation of glycogen. This literature review summarizes the development of gene therapy for the GSDs. The abnormal accumulation of glycogen and deficiency of glucose production in GSDs lead to unique symptoms based upon the enzyme step and tissues...

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PMID: 37421310
DOI: 10.1002/jimd.12654

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