Journal of Shanghai Jiao Tong University (Medical Science) >

2026 , Vol. 46 >Issue 3: 358 - 367

DOI: https://doi.org/10.3969/j.issn.1674-8115.2026.03.010

Review

Advances in CRISPR/Cas gene editing and its applications in cardiovascular diseases

  • Tian Qijia ,
  • Jia Kangni ,
  • Yan Xiaoxiang
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  • Department of Cardiovascular Medicine, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China
Yan Xiaoxiang, E-mail: cardexyanxx@hotmail.com.

Received date: 2025-07-27

  Accepted date: 2025-12-30

  Online published: 2026-03-30

Supported by

Major International (Regional) Joint Research Program of the National Natural Science Foundation of China(82120108003)

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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.

Key words： gene editing; cardiovascular disease; CRISPR/Cas

Cite this article

Tian Qijia , Jia Kangni , Yan Xiaoxiang . Advances in CRISPR/Cas gene editing and its applications in cardiovascular diseases[J]. Journal of Shanghai Jiao Tong University (Medical Science), 2026 , 46(3) : 358 -367 . DOI: 10.3969/j.issn.1674-8115.2026.03.010

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