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Gene therapy for inherited arrhythmias

Vassilios J Bezzerides1, Maksymilian Prondzynski1, Lucie Carrier2,3

  • 1Department of Cardiology, Boston Children's Hospital, 300 Longwood Ave, Boston, MA 02115, USA.

Cardiovascular Research
|April 23, 2020
PubMed

Insights

Gene therapy offers a promising new avenue for treating inherited arrhythmias, which carry a lifelong risk of sudden death. Research is exploring these targeted treatments for conditions like catecholaminergic polymorphic ventricular tachycardia and hypertrophic cardiomyopathy.

Area of Science:

  • Cardiology
  • Genetics
  • Molecular Biology

Background:

  • Inherited arrhythmias are genetic disorders increasing sudden death risk.
  • Current therapies are insufficient and do not halt disease progression.
  • Gene mutations can affect electrophysiology, cardiac structure, or both.

Purpose of the Study:

  • To provide an overview of gene therapy strategies for inherited arrhythmias.
  • To review recent gene therapy studies for specific conditions.

Main Methods:

  • Literature review of gene therapy approaches.
  • Focus on studies for catecholaminergic polymorphic ventricular tachycardia and MYBPC3-related hypertrophic cardiomyopathy.

Main Results:

  • Gene therapies show potential for molecularly targeted treatment of inherited arrhythmias.
  • Specific examples include catecholaminergic polymorphic ventricular tachycardia and hypertrophic cardiomyopathy.

Conclusions:

  • Gene therapy presents a potent, targeted option for managing inherited arrhythmias.
  • Further research is crucial for developing effective gene-based treatments.

Keywords:
Adeno-associated virusCatecholaminergic polymorphic ventricular tachycardiaGene therapyHypertrophic cardiomyopathy

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