生物起搏器的基因治疗方法。
Gene Therapy Approaches to Biological Pacemakers.
作者信息
Farraha Melad, Kumar Saurabh, Chong James, Cho Hee Cheol, Kizana Eddy
机构信息
Centre for Heart Research, the Westmead Institute for Medical Research, The University of Sydney, Sydney, NSW 2145, Australia.
Sydney Medical School, The University of Sydney, Sydney, NSW 2006, Australia.
出版信息
J Cardiovasc Dev Dis. 2018 Oct 19;5(4):50. doi: 10.3390/jcdd5040050.
Bradycardia arising from pacemaker dysfunction can be debilitating and life threatening. Electronic pacemakers serve as effective treatment options for pacemaker dysfunction. They however present their own limitations and complications. This has motivated research into discovering more effective and innovative ways to treat pacemaker dysfunction. Gene therapy is being explored for its potential to treat various cardiac conditions including cardiac arrhythmias. Gene transfer vectors with increasing transduction efficiency and biosafety have been developed and trialed for cardiovascular disease treatment. With an improved understanding of the molecular mechanisms driving pacemaker development, several gene therapy targets have been identified to generate the phenotypic changes required to correct pacemaker dysfunction. This review will discuss the gene therapy vectors in use today along with methods for their delivery. Furthermore, it will evaluate several gene therapy strategies attempting to restore biological pacing, having the potential to emerge as viable therapies for pacemaker dysfunction.
由起搏器功能障碍引起的心动过缓可能使人衰弱并危及生命。电子起搏器是治疗起搏器功能障碍的有效选择。然而，它们也有自身的局限性和并发症。这促使人们开展研究，以发现更有效、更具创新性的方法来治疗起搏器功能障碍。基因治疗因其治疗包括心律失常在内的各种心脏疾病的潜力而正在被探索。具有更高转导效率和生物安全性的基因传递载体已被开发并用于心血管疾病治疗的试验。随着对驱动起搏器发育的分子机制有了更深入的了解，已确定了几个基因治疗靶点，以产生纠正起搏器功能障碍所需的表型变化。本综述将讨论目前正在使用的基因治疗载体及其递送方法。此外，它还将评估几种试图恢复生物起搏的基因治疗策略，这些策略有可能成为治疗起搏器功能障碍的可行疗法。
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