Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia.

Abstract

Over the last three decades, the genetic basis of various inherited arrhythmia syndromes has been elucidated, providing key insights into cardiomyocyte biology and various regulatory pathways associated with cellular excitation, contraction, and repolarisation. As varying techniques to manipulate genetic sequence, gene expression, and different cellular pathways have become increasingly defined and understood, the potential to apply various gene-based therapies to inherited arrhythmia has been explored. The promise of gene therapy has generated significant interest in the medical and lay press, providing hope for sufferers of seemingly incurable disorders to imagine a future without repeated medical intervention, and, in the case of various cardiac disorders, without the risk of sudden death. In this review, we focus on catecholaminergic polymorphic ventricular tachycardia (CPVT), discussing the clinical manifestations, genetic basis, and molecular biology, together with current avenues of research related to gene therapy.

• Authors: Paloma Remior Pérez, Robyn J Hylind, Thomas M Roston, Vassilios J Bezzerides, Dominic J Abrams
• Journal: Heart, lung & circulation (Heart Lung Circ) Vol. 32 Issue 7 Pg. 790-797 (Jul 2023) ISSN: 1444-2892 [Electronic] Australia
• PMID: 37032191 (Publication Type: Review, Journal Article)
• Copyright: Copyright © 2023 Australian and New Zealand Society of Cardiac and Thoracic Surgeons (ANZSCTS) and the Cardiac Society of Australia and New Zealand (CSANZ). Published by Elsevier B.V. All rights reserved.

Chemical References

• Ryanodine Receptor Calcium Release Channel

Topics

• Humans
• Ryanodine Receptor Calcium Release Channel (genetics)
• Tachycardia, Ventricular (genetics, therapy)
• Genetic Therapy
• Myocytes, Cardiac (metabolism)
• Mutation