人类心脏基因治疗。
Human Cardiac Gene Therapy.
机构信息
From the Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY.
出版信息
Circ Res. 2018 Aug 17;123(5):601-613. doi: 10.1161/CIRCRESAHA.118.311587.
Abstract
In the past 10 years, there has been tremendous progress made in the field of gene therapy. Effective treatments of Leber congenital amaurosis, hemophilia, and spinal muscular atrophy have been largely based on the efficiency and safety of adeno-associated vectors. Myocardial gene therapy has been tested in patients with heart failure using adeno-associated vectors with no safety concerns but lacking clinical improvements. Cardiac gene therapy is adapting to the new developments in vectors, delivery systems, targets, and clinical end points and is poised for success in the near future.
摘要
在过去的 10 年中，基因治疗领域取得了巨大的进展。基于腺相关病毒载体的高效性和安全性，莱伯先天性黑蒙、血友病和脊髓性肌萎缩症的有效治疗方法已经取得了很大进展。使用腺相关病毒载体对心力衰竭患者进行心肌基因治疗，没有安全性问题，但缺乏临床改善。心脏基因治疗正在适应载体、传递系统、靶点和临床终点的新发展，并准备在不久的将来取得成功。
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