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基于诱导多能干细胞的遗传性心律失常治疗:相关机遇与挑战(综述)

Induced pluripotent stem cell-based therapies for inherited arrhythmias: opportunities and challenges involved (Review).

作者信息

Li Guoliang, He Xin, Sun Chaofeng

机构信息

Department of Cardiovascular Medicine, The First Affiliated Hospital of Xi'an Jiaotong University College of Medicine, Xi'an, Shaanxi 710061, P.R. China.

出版信息

Mol Med Rep. 2015 Jan;11(1):3-10. doi: 10.3892/mmr.2014.2668. Epub 2014 Oct 16.

DOI:10.3892/mmr.2014.2668
PMID:25323841
Abstract

The identification of induced pluripotent stem cell (iPSC) technology represents great potential for recapitulating complex physiological phenotypes, probing toxicological testing and screening candidate drugs, demonstrating novel mechanistic insights and, in particular, applying iPSC-based therapeutic strategies for inherited disorders. Inherited arrhythmias are caused by various genetic abnormalities and harbor similar clinical outcomes. Clinically, the poorest outcomes are fatal arrhythmias and sudden cardiac death. However, the current therapeutic options for inherited arrhythmias are inadequate and problematic. In this review, we summarize the advances of the iPSC technique in the field of inherited arrhythmias and discuss the possibility of iPSC‑based therapies for inherited arrhythmias. Additionally, we highlight the key challenges faced in the field of iPSC and the emerging strategies used to address these concerns before the novel technique can be used safely and efficiently in clinical practice. It is likely that the iPSC technique will present opportunities and further challenges in the future.

摘要

诱导多能干细胞(iPSC)技术的发现为重现复杂的生理表型、探索毒理学测试和筛选候选药物、揭示新的机制见解,特别是将基于iPSC的治疗策略应用于遗传性疾病展现出了巨大潜力。遗传性心律失常由各种基因异常引起,并具有相似的临床结果。临床上,最糟糕的结果是致命性心律失常和心源性猝死。然而,目前针对遗传性心律失常的治疗选择并不充分且存在问题。在本综述中,我们总结了iPSC技术在遗传性心律失常领域的进展,并讨论了基于iPSC的遗传性心律失常治疗方法的可能性。此外,我们强调了iPSC领域面临的关键挑战以及在这项新技术能够在临床实践中安全有效地应用之前用于解决这些问题的新兴策略。iPSC技术未来可能会带来机遇和更多挑战。

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