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基于人类诱导多能干细胞和纳米技术的疗法。

Human induced pluripotent stem cell and nanotechnology-based therapeutics.

作者信息

Liu Wei-Hsiu, Chang Yuh-Lih, Lo Wen-Liang, Li Hsin-Yang, Hsiao Chia-Wei, Peng Chi-Hsien, Chiou Shih-Hwa, Ma Hsin-I, Chen Shih-Jen

机构信息

Graduate Institute of Medical Sciences, National Defense Medical Center, Taipei, Taiwan, ROC.

出版信息

Cell Transplant. 2015;24(11):2185-95. doi: 10.3727/096368914X685113. Epub 2014 Oct 8.

Abstract

Human induced pluripotent stem cells (hiPSCs) can be genetically reprogrammed to an embryonic stem cell-like state and can provide promising medical applications, such as diagnosis, prognosis, drug screening for therapeutical development, and monitoring disease progression. Despite myriad advances, traditional viral-based reprogramming for generating hiPSCs has safety risks that hinder further practical applications of hiPSCs. In the past decade, nonviral-based reprogramming has been used as an alternative to produce hiPSCs and enhance their differentiation. In addition, the efficiency of nonviral-based reprogramming is generally poor, compared to that of viral-based reprogramming. Recent studies in nanoscale-structured particles have made progress in addressing many applications of hiPSCs for clinical practice. The combination of hiPSCs and nanotechnology will actually act as the therapeutic platform for personalized medicine and can be the remedies against various diseases in the future. In this article, we review recent advances in cellular reprogramming and hiPSC-related research, such as cell source, delivery system, and direct reprogramming, as well as some of its potential clinical applications, including mitochondrial and retinal disease. We also briefly summarize the current incorporation of nanotechnology in patient-specific hiPSCs for future treatments.

摘要

人诱导多能干细胞(hiPSC)可以通过基因重编程转变为胚胎干细胞样状态,并具有广阔的医学应用前景,如诊断、预后评估、药物筛选以促进治疗药物开发以及监测疾病进展等。尽管取得了众多进展,但传统的基于病毒的重编程方法来生成hiPSC存在安全风险,这阻碍了hiPSC的进一步实际应用。在过去十年中,基于非病毒的重编程方法已被用作生成hiPSC并促进其分化的替代方法。此外,与基于病毒的重编程相比,基于非病毒的重编程效率通常较低。最近关于纳米级结构颗粒的研究在解决hiPSC在临床实践中的许多应用方面取得了进展。hiPSC与纳米技术的结合实际上将成为个性化医学的治疗平台,并可能在未来成为对抗各种疾病的疗法。在本文中,我们综述了细胞重编程和hiPSC相关研究的最新进展,如细胞来源、递送系统和直接重编程,以及其一些潜在的临床应用,包括线粒体疾病和视网膜疾病。我们还简要总结了目前纳米技术在患者特异性hiPSC中的应用情况,以供未来治疗参考。

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