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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Publicat a:Mol Ther Methods Clin Dev
Autors principals: Yang, Chun, Tian, Wenhong, Ma, Sisi, Guo, Mengmeng, Lin, Xiao, Gao, Fengying, Dong, Xiaoyan, Gao, Mingming, Wang, Yuhui, Liu, George, Xian, Xunde
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7424175/
https://ncbi.nlm.nih.gov/pubmed/32802915
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.07.011
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