Article
[The safety and efficacy of adeno-associated virus-mediated LDLR transfection in homozygous familial hypercholesterolemia].
Zhonghua xin xue guan bing za zhi - 24 Mar 2026
Gao G, Xu C B, Li R F, Zheng T, Shao J R, Wang X K, Liu P N, Wang G, Jiang L X, Qu G, Chen T, She J Q, Luo Y B, Yuan Z Y, Wu Y
Abstract excerpt
Objective: To evaluate the safety and efficacy of an adeno-associated virus vector carrying an optimized human low density lipoprotein receptor (LDLR) gene (NGGT006) in the treatment of homozygous familial hypercholesterolemia (HoFH) with LDLR mutations. Methods: This is an open-label, single-center, single-arm, non-randomized, investigator-initiated clinical trial. According to the dose-escalation principle,...
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