Department of Medicine, University of California San Diego, and VA San Diego Healthcare System, San Diego, Calif., USA.
Transl Res. 2013 Apr;161(4):313-20. doi: 10.1016/j.trsl.2012.11.004. Epub 2012 Dec 20.
Congestive heart failure is a major cause of morbidity and mortality with increasing social and economic costs. There have been no new high impact therapeutic agents for this devastating disease for more than a decade. However, many pivotal regulators of cardiac function have been identified using cardiac-directed transgene expression and gene deletion in preclinical studies. Some of these increase function of the failing heart. Altering the expression of these pivotal regulators using gene transfer is now either being tested in clinical gene transfer trials, or soon will be. In this review, we summarize recent progress in cardiac gene transfer for clinical congestive heart failure.
充血性心力衰竭是发病率和死亡率的主要原因，社会和经济成本不断增加。十多年来，这种破坏性疾病没有新的高影响力治疗药物。然而，通过心脏定向转基因表达和基因缺失的临床前研究，已经确定了许多心脏功能的关键调节因子。其中一些增加了衰竭心脏的功能。目前正在进行或即将进行使用基因转移改变这些关键调节因子表达的临床基因转移试验。在这篇综述中，我们总结了充血性心力衰竭的临床心脏基因转移的最新进展。
