Cardiogenetics (Aug 2024)

Gene Therapy for Inherited Arrhythmia Syndromes

  • Cameron J. Leong,
  • Sohat Sharma,
  • Jayant Seth,
  • Archan Dave,
  • Abdul Aziz Abdul Ghafoor,
  • Zachary Laksman

DOI
https://doi.org/10.3390/cardiogenetics14030011
Journal volume & issue
Vol. 14, no. 3
pp. 132 – 148

Abstract

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The emergence of gene therapy offers opportunities for treating a myriad of genetic disorders and complex diseases that previously had limited or no treatment options. The key basic strategies for gene therapy involve either the addition, inhibition, or introduction of a new gene, with a crucial component being the use of a delivery vector to effectively target cells. Particularly promising is the application of gene therapy for the treatment of inherited arrhythmia syndromes, conditions associated with significant mortality and morbidity that have limited treatment options, and a paucity of disease modifying therapy. This review aims to summarize the utility of gene therapy for the treatment of inherited arrhythmia syndromes by exploring the current state of knowledge, limitations, and future directions.

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