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Viral Transgene Expression in Rodent Hearts and the Assessment of Cardiac Arrhythmia Risk
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Gene Therapy for Inherited Cardiac Arrhythmias

Mara R Turkieltaub Paredes1,2, Mohit Hulsurkar1,2, Xander H T Wehrens3,4,5,6,7,8

  • 1Cardiovascular Research Institute, Baylor College of Medicine, Houston, TX, 77030, USA.

Journal of Cardiovascular Translational Research
|October 30, 2025
PubMed

Insights

Gene therapy offers a promising, potentially curative treatment for inherited cardiac arrhythmias, which are genetic heart rhythm disorders. This approach addresses limitations of conventional treatments and explores gene delivery advancements for conditions like Brugada syndrome.

Area of Science:

  • Cardiology
  • Genetics
  • Molecular Biology

Background:

  • Inherited cardiac arrhythmias are genetic disorders affecting the heart's electrical system.
  • These conditions significantly increase mortality risk due to inherited mutations.
  • Current treatments like drugs and surgery often lack efficacy and cause complications.

Purpose of the Study:

  • To review gene therapy applications for inherited cardiac arrhythmias.
  • To analyze gene delivery methods for treating these heart rhythm disorders.
  • To highlight the therapeutic potential and challenges of gene therapy.

Main Methods:

  • Review of recent studies on gene therapy for specific inherited arrhythmias.
  • Analysis of advancements in viral and non-viral gene delivery systems.
  • Examination of molecular mechanisms and candidate genes.

Main Results:

  • Gene therapy is emerging as a feasible and potentially curative treatment.
  • Elucidation of molecular mechanisms and identification of candidate genes are advancing.
  • Progress in gene delivery methods enhances therapeutic potential.

Conclusions:

  • Gene therapy presents a viable alternative to conventional treatments for inherited cardiac arrhythmias.
  • Further advancements in gene delivery are crucial for therapeutic success.
  • This review covers Brugada syndrome, CPVT, LQTS, AF, and ACM, assessing gene therapy's role.

Keywords:
ArrhythmiasArrhythmogenic cardiomyopathyAtrial fibrillationBrugada syndromeCatecholaminergic polymorphic ventricular tachycardiaGene therapyLong QT syndromeViral vectors

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