Cardiovascular Research Center, Mount Sinai School of Medicine, New York, NY, USA.
J Am Coll Cardiol. 2011 Mar 8;57(10):1169-80. doi: 10.1016/j.jacc.2010.11.023.
Congestive heart failure is a major cause of morbidity and mortality in the United States. Although progress in conventional treatments is making steady and incremental gains to decrease heart failure mortality, there is a critical need to explore new therapeutic approaches. Gene therapy was initially applied in the clinical setting for inherited monogenic disorders. It is now apparent that gene therapy has broader potential that also includes acquired polygenic diseases, such as congestive heart failure. Recent advances in understanding of the molecular basis of myocardial dysfunction, together with the evolution of increasingly efficient gene transfer technology, have placed heart failure within the reach of gene-based therapy. Furthermore, the recent successful and safe completion of a phase 2 trial targeting the sarcoplasmic reticulum Ca(2+) ATPase pump along with the start of more recent phase 1 trials are ushering in a new era of gene therapy for the treatment of heart failure.
充血性心力衰竭是美国发病率和死亡率的主要原因。尽管在常规治疗方面取得了稳步和渐进的进展，以降低心力衰竭死亡率，但仍迫切需要探索新的治疗方法。基因治疗最初应用于临床遗传性单基因疾病。现在很明显，基因治疗具有更广泛的潜力，也包括获得性多基因疾病，如充血性心力衰竭。对心肌功能障碍分子基础的理解的最新进展，以及越来越高效的基因转移技术的发展，使心力衰竭成为基因治疗的目标。此外，最近针对肌浆网 Ca（2+）ATP 酶泵的 2 期试验的成功和安全完成，以及最近开始的 1 期试验，为心力衰竭的基因治疗开辟了一个新时代。
