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

IF 3.1 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Current Cardiology Reports Pub Date : 2024-09-01 Epub Date: 2024-08-07 DOI:10.1007/s11886-024-02096-5

Jamie R Johnston, Eric D Adler

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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.

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精准基因疗法：平衡心力衰竭患者的风险与收益。

综述的目的：精准基因医学正在飞速发展，并对临床心脏病学产生了重大影响。在此，我们将讨论心肌病和心力衰竭基因疗法的最新进展和新兴策略：阐明心力衰竭的基因结构为心血管医学中的精准疗法铺平了道路。最近的临床前研究和早期临床试验取得了令人鼓舞的成果，为开发针对各种病因引起的心力衰竭的基因疗法提供了支持。除了发现新的治疗靶点，创新的给药平台也被用来提高心脏基因疗法的安全性和有效性。精准基因疗法是改善心衰患者预后的一种潜在安全有效的方法。它直接针对潜在病因，有望从根本上改变心衰的治疗模式。随着新一代心血管药物进入临床，仔细评估对患者的益处和风险尤为重要。

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来源期刊

Current Cardiology Reports CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

6.20

自引率

2.70%

发文量

209

期刊介绍： The aim of this journal is to provide timely perspectives from experts on current advances in cardiovascular medicine. We also seek to provide reviews that highlight the most important recently published papers selected from the wealth of available cardiovascular literature. We accomplish this aim by appointing key authorities in major subject areas across the discipline. Section editors select topics to be reviewed by leading experts who emphasize recent developments and highlight important papers published over the past year. An Editorial Board of internationally diverse members suggests topics of special interest to their country/region and ensures that topics are current and include emerging research. We also provide commentaries from well-known figures in the field.