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Gene Therapy for Cardiovascular and Cerebrovascular Disease: Mechanisms, Translational Barriers, and the Road Ahead

Zixu Liu1, Ruiqi Liu1, Ying Ying2

  • 1Queen Mary College, Jiangxi Medical College, Nanchang University, Nanchang 330006, China.

Biomedicines
|May 27, 2026
PubMed

Insights

Gene therapy offers new hope for cardiovascular and cerebrovascular diseases like arrhythmias, atherosclerosis, and stroke. These advanced treatments target root causes, potentially transforming patient outcomes beyond conventional care.

Area of Science:

  • Cardiovascular and cerebrovascular research
  • Genetic medicine
  • Translational science

Background:

  • Cardiovascular and cerebrovascular diseases are leading causes of death and disability worldwide.
  • Conventional therapies leave significant residual risk, necessitating novel interventions.
  • Gene-based approaches offer potential to address underlying molecular pathology.

Purpose of the Study:

  • To review current gene therapy strategies for cardiovascular and cerebrovascular diseases.
  • To synthesize mechanistic understanding, translational obstacles, and future directions.
  • To evaluate gene therapy's potential to complement standard care.

Main Methods:

  • Review of gene-based interventions including KCNQ1/KCNH2 suppression, SCN5A base editing, PKP2/TMEM43 restoration for arrhythmias.
  • Analysis of RNA-based agents (inclisiran) and in vivo editing (VERVE-101) for atherosclerosis.
  • Examination of cGAS-STING silencing, AAV-NeuroD1 reprogramming, and neurotrophic factor delivery for ischemic stroke.

Main Results:

  • Gene therapies show promise in stabilizing electrophysiology, reducing lipids, and attenuating vascular inflammation.
  • Specific strategies like AAV-NeuroD1 and neurotrophic factors extend therapeutic windows post-stroke.
  • These approaches address fundamental molecular causes, offering a new therapeutic paradigm.

Conclusions:

  • Gene therapy presents a meaningful complement to existing treatments for cardiovascular and cerebrovascular diseases.
  • Despite delivery and safety challenges, gene therapy is poised to significantly impact disease prevention and treatment.
  • Addressing root molecular pathology offers a transformative potential for managing these conditions.

Keywords:
atherosclerosiscardiac arrhythmiacardiovascular diseasecerebrovascular diseasegene therapyischaemic stroke

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