Research Progress on Animal Models and Treatments Related to GSD-1a (Glycogen Storage Disease)
WANG Xiaoyi, GE Mengting, XIE Yixia, YAN Junyan, JIN Lifang*
GSD-1a (glycogen storage disease type 1a) is an autosomal recessive metabolic disease caused by glucose-6-phosphatase-α (G6Pas-α or G6PC) deficiency. The clinical manifestations are liver and kidney dysfunction with high mortality. The recent establishment of large animal models of GSD-1a based on new gene editing technology and more efficient gene therapy methods will become a new and important research trend. This article focuses on current research progress in GSD-1a as well as its underlying mechanisms and potential treatments based on molecular genetics, animal model establishment and application, and drug and gene therapy.
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