The present and future role of ultrasound targeted microbubble destruction in preclinical studies of cardiac gene therapy

超声靶向微泡破坏在心脏基因治疗临床前研究中的现状和未来作用

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作者:Lijun Qian, Barsha Thapa, Jian Hong, Yanmei Zhang, Menglin Zhu, Ming Chu, Jing Yao, Di Xu

Abstract

Multiple limitations for cardiac pharmacologic therapies like intolerance, individual variation in effectiveness, side effects, and high cost still remain, despite the recent progress in diagnosis and health support. Gene therapy is poised to be an attractive alternative in various ways for the future, refractory cardiac diseases being one aspect of it. As a novel therapy to deliver the objective gene to organs of living animals, ultrasound targeted microbubble destruction (UTMD) has therapeutic potential in cardiovascular disorders. UTMD, which binds microbubbles with DNA or RNA carriers into the shell and destroys the located microbubbles with low frequency and high mechanical index ultrasound can release target agents to specific organs. UTMD has the ability to transfect markedly through sonoporation, cavitation and other effects by way of intravenous injection that is minimally invasive and highly specific for gene deliverance. Here, we have summarized the present role of UTMD in pre-clinical studies of cardiac gene therapy which covers myocardial infarction, regeneration, ischaemia/reperfusion injury, hypertension, diabetic cardiomyopathy, adriamycin cardiomyopathy and some discussion for further studies.

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