In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial

Nature Medicine 2026

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Authors: Ping Wan, Siyuan Tang, Dongni Lin, Yuming Lu, Mei Long, Ling Xiao, Yanhong Jiang, Jiaoyang Liao, Xiaoying Ma, Ying Liu, Wensu Yu, Michael Ott
+4 more Zi Jun Wang, Yuxuan Wu, Taihua Yang, Qiang Xia
DOI: 10.1038/s41591-026-04254-4
Cited by: 22
References: 39

Fields of Study & Subjects

Virus-based gene therapy research Lipoproteins and Cardiovascular Health CRISPR and Genetic Engineering

Publication details

Journal / source
Nature Medicine
Publisher
Springer Science and Business Media LLC
Volume
32
Issue
3
Pages
1045–1051
Publication date
1 March 2026
Language
EN