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Combining Induced Pluripotent Stem Cells and Genome Editing Technologies for Clinical Applications

机译:结合诱导多能干细胞和基因组编辑技术对临床应用

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摘要

In this review, we introduce current developments in induced pluripotent stem cells (iPSCs), site-specific nuclease (SSN)-mediated genome editing tools, and the combined application of these two novel technologies in biomedical research and therapeutic trials. The sustainable pluripotent property of iPSCs in vitro not only provides unlimited cell sources for basic research but also benefits precision medicines for human diseases. In addition, rapidly evolving SSN tools efficiently tailor genetic manipulations for exploring gene functions and can be utilized to correct genetic defects of congenital diseases in the near future. Combining iPSC and SSN technologies will create new reliable human disease models with isogenic backgrounds in vitro and provide new solutions for cell replacement and precise therapies.
机译:在本文中,我们在诱导多能干细胞(IPSC),位点特异性核酸酶(SSN)介导的基因组编辑工具中引入电流发展,以及这两种新技术在生物医学研究和治疗试验中的组合应用。 IPSCS在体外的可持续多能性质不仅为基础研究提供无限制的细胞来源,而且为人类疾病的精密药物效益。 此外,快速发展的SSN工具有效地定制了用于探索基因功能的遗传操作,可用于在不久的将来纠正先天性疾病的遗传缺陷。 组合IPSC和SSN技术将创造新的可靠的人类疾病模型,体外具有均匀性背景,为细胞更换和精确疗法提供新的解决方案。

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