AAV-Mediated ApoC2 Gene Therapy: Reversal of Severe Hypertriglyceridemia and Rescue of Neonatal Death in ApoC2-Deficient Hamsters

AAV-Mediated ApoC2 Gene Therapy: Reversal of Severe Hypertriglyceridemia and Rescue of Neonatal Death in ApoC2-Deficient Hamsters

Apolipoprotein C2 (ApoC2) is a key activator of lipoprotein lipase for plasma triglyceride metabolism. ApoC2-deficient patients present with severe hypertriglyceridemia and recurrent acute pancreatitis, for whom the only effective treatment is the infusion of normal plasma containing ApoC2. However,...

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Bibliographic Details
Main Authors: Chun Yang, Wenhong Tian, Sisi Ma, Mengmeng Guo, Xiao Lin, Fengying Gao, Xiaoyan Dong, Mingming Gao, Yuhui Wang, George Liu, Xunde Xian
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301601

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http://www.sciencedirect.com/science/article/pii/S2329050120301601

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