Hayward Carl, Banner Nicholas R, Morley-Smith Andrew, Lyon Alexander R, Harding Sian E
1Cardiovascular Biomedical Research Unit, Royal Brompton Hospital, SW3 6NP London, United Kingdom.
2Royal Brompton and Harefield NHS Trust, Harefield Hospital, UB9 6JH Harefield, United Kingdom.
Hum Gene Ther. 2015 May;26(5):293-304. doi: 10.1089/hum.2015.018.
Gene therapy has been applied to cardiovascular disease for over 20 years but it is the application to heart failure that has generated recent interest in clinical trials. There is laboratory and early clinical evidence that delivery of sarcoplasmic reticulum calcium ATPase 2a (SERCA2a) gene therapy is beneficial for heart failure and this therapy could become the first positive inotrope with anti-arrhythmic properties. In this review we will discuss the rationale for SERCA2a gene therapy as a viable strategy in heart failure, review the published data, and discuss the ongoing clinical trials, before concluding with comments on the future challenges and potential for this therapy.
基因治疗应用于心血管疾病已有20多年,但将其应用于心力衰竭才在近期引发了临床试验的兴趣。有实验室和早期临床证据表明,肌浆网钙ATP酶2a(SERCA2a)基因治疗对心力衰竭有益,这种治疗可能成为首个具有抗心律失常特性的正性肌力药物。在本综述中,我们将讨论SERCA2a基因治疗作为心力衰竭可行策略的理论依据,回顾已发表的数据,并讨论正在进行的临床试验,最后对该治疗的未来挑战和潜力发表评论。