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

作者：Ping Wan, Siyuan Tang, Dongni Lin, Yuming Lu, Mei Long, Ling Xiao, Yanhong Jiang, Jiaoyang Liao, Xiaoying Ma, Ying Liu, Wensu Yu, Michael Ott, Zi Jun Wang, Yuxuan Wu, Taihua Yang, Qiang Xia · 发表于：Nature Medicine · 年份：2026 · DOI：10.1038/s41591-026-04254-4 · 被引用次数：18 · 研究领域：Virus-based gene therapy research、Lipoproteins and Cardiovascular Health、CRISPR and Genetic Engineering