AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial

Nature Medicine 2026

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Authors: Tao Zheng, Ge Gao, C B Xu, R F Li, Jinrui Shao, Xiaoke Wang, Xiu Han, Juanli Li, P N Liu, Ying Xiong, Lailing Song, Yi-Xiang Wang

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Shengmei Wen, Xiaozhen Zhuo, Gang Wang, Lixin Jiang, Guang Qu, Tao Chen, Jianqing She, Yongbai Luo, Z Y Yuan, Yue Wu

DOI: 10.1038/s41591-026-04441-3

Cited by: 1

References: 40

Fields of Study & Subjects

Lipoproteins and Cardiovascular Health Virus-based gene therapy research Blood disorders and treatments

References 40