Why the study?
Precision genetic medicine is rapidly evolving with significant implications for cardiology, motivating a review of the latest advancements, emerging strategies, and the balance of benefits and risks in gene therapy for cardiomyopathy and heart failure.
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Precision genetic therapies, including gene transfer and genome editing, hold promise for transforming the treatment paradigm of heart failure by directly targeting underlying genetic etiologies.
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.
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Johnston et al. (2024) studied this question.
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