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Review
. 2024 Sep;26(9):973-983.
doi: 10.1007/s11886-024-02096-5. Epub 2024 Aug 7.

Precision Genetic Therapies: Balancing Risk and Benefit in Patients with Heart Failure

Jamie R Johnston  1 , Eric D Adler  2
Affiliations
Review

Precision Genetic Therapies: Balancing Risk and Benefit in Patients with Heart Failure

Jamie R Johnston et al. Curr Cardiol Rep. 2024 Sep.

Abstract

Purpose of review: Precision genetic medicine is evolving at a rapid pace and bears significant implications for clinical cardiology. Herein, we discuss the latest advancements and emerging strategies in gene therapy for cardiomyopathy and heart failure.

Recent findings: Elucidating the genetic architecture of heart failure has paved the way for precision therapies in cardiovascular medicine. Recent preclinical studies and early-phase clinical trials have demonstrated encouraging results that support the development of gene therapies for heart failure arising from a variety of etiologies. In addition to the discovery of new therapeutic targets, innovative delivery platforms are being leveraged to improve the safety and efficacy of cardiac gene therapies. Precision genetic therapy represents a potentially safe and effective approach for improving outcomes in patients with heart failure. It holds promise for radically transforming the treatment paradigm for heart failure by directly targeting the underlying etiology. As this new generation of cardiovascular medicines progress to the clinic, it is especially important to carefully evaluate the benefits and risks for patients.

Keywords: Cardiomyopathy; Gene Editing; Gene Therapy; Gene Transfer; Heart Failure; Muscular Dystrophy.

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Conflict of interest statement

Jamie Johnston was formerly employed at Kate Therapeutics and reports owning stock or stock options. Eric Adler is the Chief Scientific Officer at Lexeo Therapeutics and a shareholder in Rocket Pharmaceuticals. He also reports the following: consulting fees from Abiomed and Abbott; payment for expert testimony from AstraZeneca; participation on a Data Safety Monitoring Board or Advisory Board for Rocket Pharmaceuticals, Cytokinetics, and Kiniska; leadership or fiduciary role in Lexeo Therapeutics, Heart Failure Society of America, and American Heart Association; and stock or stock options for Lexeo Therapeutics, Rocket Pharmaceuticals, and Papillion Therapeutics.

Jamie Johnston was formerly employed at Kate Therapeutics and reports owning stock or stock options. Eric Adler is the Chief Scientific Officer at Lexeo Therapeutics and a shareholder in Rocket Pharmaceuticals. He also reports the following: consulting fees from Abiomed and Abbott; payment for expert testimony from AstraZeneca; participation on a Data Safety Monitoring Board or Advisory Board for Rocket Pharmaceuticals, Cytokinetics, and Kiniska; leadership or fiduciary role in Lexeo Therapeutics, Heart Failure Society of America, and American Heart Association; and stock or stock options for Lexeo Therapeutics, Rocket Pharmaceuticals, and Papillion Therapeutics.

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