Oh Jae Gyun, Ishikawa Kiyotake
Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Corresponding author.
Discov Med. 2019 Nov-Dec;28(155):229-235.
There has been significant progress in the field of gene therapy toward clinical application in recent years. Cardiac gene therapy has followed this trend, but at a slower pace compared to therapies targeting retinal, spinal, and blood disorders. New approaches targeting monogenic disorders are being developed at a rapid pace and studies in large animal models show promise. Meanwhile, several clinical trials are underway to prove the efficacy of gene therapy for cardiovascular diseases such as heart failure. In this concise review, we attempt to summarize recent highlights in technological advancements, preclinical studies, and clinical trials of cardiac gene therapy.
近年来,基因治疗领域在临床应用方面取得了重大进展。心脏基因治疗也顺应了这一趋势,但与针对视网膜、脊髓和血液疾病的治疗相比,其发展速度较慢。针对单基因疾病的新方法正在迅速开发,在大型动物模型中的研究显示出了前景。与此同时,多项临床试验正在进行,以证明基因治疗对心力衰竭等心血管疾病的疗效。在这篇简要综述中,我们试图总结心脏基因治疗在技术进步、临床前研究和临床试验方面的近期亮点。