上海交通大学学报（医学版）2026，Vol.46Issue(3)：358-367,10.DOI:10.3969/j.issn.1674-8115.2026.03.010
CRISPR/Cas基因编辑技术在心血管疾病中的应用研究进展
Advances in CRISPR/Cas gene editing and its applications in cardiovascular diseases
摘要
Abstract
Gene editing technologies refer to a class of techniques capable of precisely modifying or manipulating the genome or transcriptome of living organisms.Over the past decade,next-generation gene editing tools,represented by CRISPR/Cas9,have rapidly advanced,demonstrating superior efficiency,safety,and broader applicability compared to traditional methods,such as zinc finger nuclease(ZFN)and transcription activator-like effector nuclease(TALEN).These advancements have facilitated the transition of gene editing from basic research to clinical applications.Cardiovascular diseases remain a leading cause of global mortality and health burden,yet current treatments face challenges such as undruggable targets,significant side effects,and limited efficacy.For certain conditions that are difficult to treat with conventional pharmacotherapy,gene editing offers a novel therapeutic approach.Currently,this technology has been successfully applied to drug development for diseases such as transthyretin amyloidosis and hyperlipidemia,with pioneering candidates such as NTLA-2001 and CTX310 having entered clinical trials.Mitigating immune responses and off-target effects,along with overcoming cardiac-targeted delivery barriers,are pivotal for clinical translation.With advancements in artificial intelligence-assisted design and non-viral vectors,CRISPR/Cas technology is expected to overcome current liver-predominant limitations,paving the way for in situ cardiac treatment and offering a curative strategy for cardiovascular diseases.This article systematically reviews the structure and mechanism of CRISPR/Cas systems and summarizes recent advances in their applications to cardiovascular diseases,including gene knockout,epigenetic editing,base editing,prime editing,and RNA editing,aiming to provide important references for subsequent research and clinical translation in this field.关键词
基因编辑/心血管疾病/CRISPR/CasKey words
gene editing/cardiovascular disease/CRISPR/Cas分类
医药卫生引用本文复制引用
田启嘉,贾康妮,闫小响..CRISPR/Cas基因编辑技术在心血管疾病中的应用研究进展[J].上海交通大学学报（医学版）,2026,46(3):358-367,10.基金项目
国家自然科学基金重点国际合作项目(82120108003). Major International(Regional)Joint Research Program of the National Natural Science Foundation of China(82120108003). （82120108003）
