精准基因治疗：心力衰竭患者的风险与获益平衡。
Precision Genetic Therapies: Balancing Risk and Benefit in Patients with Heart Failure.
机构信息
Department of Biomedical Sciences, College of Medicine, Florida State University, Tallahassee, FL, 32306, USA.
Division of Cardiology, Department of Internal Medicine, University of California San Diego, La Jolla, CA, 92037, USA.
出版信息
Curr Cardiol Rep. 2024 Sep;26(9):973-983. doi: 10.1007/s11886-024-02096-5. Epub 2024 Aug 7.
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.
目的综述
精准遗传学发展迅速，对临床心脏病学具有重要意义。本文讨论了心肌病和心力衰竭基因治疗的最新进展和新兴策略。
最近发现
阐明心力衰竭的遗传结构为心血管医学的精准治疗铺平了道路。最近的临床前研究和早期临床试验结果令人鼓舞，支持针对多种病因的心力衰竭的基因治疗发展。除了发现新的治疗靶点外，还利用创新的传递平台来提高心脏基因治疗的安全性和有效性。精准基因治疗为改善心力衰竭患者的预后提供了一种潜在的安全有效的方法。通过直接针对潜在病因，为心力衰竭的治疗模式带来了彻底变革的希望。随着新一代心血管药物进入临床，仔细评估患者的获益和风险尤为重要。
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