Why the study?
ApoC2 deficiency causes severe hypertriglyceridemia, and current treatment requires repeated plasma infusions due to rapid ApoC2 catabolism, limiting clinical utility.
Does AAV-hApoC2 gene therapy improve severe hypertriglyceridemia and prevent neonatal death in ApoC2-deficient hamsters?
Does AAV-hApoC2 gene therapy improve severe hypertriglyceridemia and prevent neonatal death in ApoC2-deficient hamsters?
AAV-mediated ApoC2 gene therapy is a promising approach for treating severe hypertriglyceridemia caused by ApoC2 deficiency, based on successful rescue in a hamster model.
Answers are grounded in this paper and cite their sources.
Supports AAV-hApoC2 development for ApoC2 deficiency; leaves open human translation in severe hypertriglyceridemia.
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, since ApoC2 has a fast catabolic rate, a repeated infusion is required, which limits its clinical use. To explore a safe and efficient approach for ApoC2 deficiency, we herein established an adeno-associated virus expressing human ApoC2 (AAV-hApoC2) to evaluate the efficacy and safety of gene therapy in ApoC2-deficient hypertriglyceridemic hamsters. Administration of AAV-hApoC2 via jugular or orbital vein in adult and neonatal ApoC2-deficient hamsters, respectively, could prevent the neonatal death and effectively improve severe hypertriglyceridemia of ApoC2-deficient hamsters without side effects in a long-term manner. Our novel findings in the present study demonstrate that AAV-hApoC2-mediated gene therapy will be a promising therapeutic approach for clinical patients with severe hypertriglyceridemia caused by ApoC2 deficiency.
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Yang et al. (2020) studied this question.
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