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Gene therapy for glycogen storage diseases

7 Jun 2019

Abstract excerpt

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 example, glucose-6-phosphatase (G6Pase) deficiency in GSD type Ia (GSD Ia) affects primarily the liver and kidney,...

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PMID: 31227835
DOI: 10.1093/hmg/ddz133

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