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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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| Udgivet i: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
American Society of Gene & Cell Therapy
2020
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| Fag: | |
| Online adgang: | 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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