中文内容
已翻译research literature英文原文2026-06-05
期刊：Nature Medicine；在线发表：2026 年 6 月 4 日；DOI：10.1038/s41591-026-04441-3
After preclinical testing, a phase 1 trial involving three individuals with familial hypercholesterolemia demonstrated preliminary evidence for the safety and efficacy of AAV8-mediated LDL receptor gene therapy.