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Induced pluripotent stem cell-based therapies for inherited arrhythmias: Opportunities and challenges involved

机译:诱导性多能干细胞疗法治疗遗传性心律失常:涉及的机遇和挑战

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摘要

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.
机译:诱导多能干细胞(iPSC)技术的鉴定代表了概括复杂生理表型,探查毒理学测试和筛选候选药物,展示新颖的机理见解,特别是将基于iPSC的治疗策略应用于遗传性疾病的巨大潜力。遗传性心律失常是由各种遗传异常引起的,具有相似的临床结果。临床上,最差的结果是致命性心律失常和心脏猝死。然而,当前用于遗传性心律不齐的治疗选择是不足的和成问题的。在这篇综述中,我们总结了iPSC技术在遗传性心律失常领域中的进展,并讨论了基于iPSC的遗传性心律失常疗法的可能性。此外,我们重点介绍了iPSC领域面临的主要挑战以及在安全有效地将新技术用于临床实践之前用于解决这些问题的新兴策略。 iPSC技术可能会在将来带来机遇和进一步的挑战。

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