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干细胞:人类心脏疾病建模和再生医学的变革者。

Stem Cells: The Game Changers of Human Cardiac Disease Modelling and Regenerative Medicine.

机构信息

Department of Experimental and Clinical Medicine, Research Center for Advanced Biochemistry and Molecular Biology, University "Magna Graecia" of Catanzaro, 88100 Loc., Germaneto, Catanzaro, Italy.

出版信息

Int J Mol Sci. 2019 Nov 16;20(22):5760. doi: 10.3390/ijms20225760.

Abstract

A comprehensive understanding of the molecular basis and mechanisms underlying cardiac diseases is mandatory for the development of new and effective therapeutic strategies. The lack of appropriate cell models that faithfully mirror the human disease phenotypes has hampered the understanding of molecular insights responsible of heart injury and disease development. Over the past decade, important scientific advances have revolutionized the field of stem cell biology through the remarkable discovery of reprogramming somatic cells into induced pluripotent stem cells (iPSCs). These advances allowed to achieve the long-standing ambition of modelling human disease in a dish and, more interestingly, paved the way for unprecedented opportunities to translate bench discoveries into new therapies and to come closer to a real and effective stem cell-based medicine. The possibility to generate patient-specific iPSCs, together with the new advances in stem cell differentiation procedures and the availability of novel gene editing approaches and tissue engineering, has proven to be a powerful combination for the generation of phenotypically complex, pluripotent stem cell-based cellular disease models with potential use for early diagnosis, drug screening, and personalized therapy. This review will focus on recent progress and future outcome of iPSCs technology toward a customized medicine and new therapeutic options.

摘要

全面了解心脏疾病的分子基础和机制对于开发新的有效治疗策略是必要的。缺乏能够真实反映人类疾病表型的合适细胞模型,阻碍了对导致心脏损伤和疾病发展的分子机制的理解。在过去的十年中,通过对体细胞进行重编程为诱导多能干细胞(iPSC)的惊人发现,重要的科学进展彻底改变了干细胞生物学领域。这些进展使人们能够实现长期以来在培养皿中模拟人类疾病的目标,更有趣的是,为将实验室发现转化为新疗法并更接近真正有效的基于干细胞的医学铺平了道路。生成患者特异性 iPSC 的可能性,以及干细胞分化程序的新进展和新型基因编辑方法和组织工程的应用,已被证明是生成具有潜在用于早期诊断、药物筛选和个性化治疗的表型复杂、多能干细胞为基础的细胞疾病模型的有力组合。这篇综述将重点介绍 iPSC 技术在定制医学和新治疗选择方面的最新进展和未来前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6a93/6888119/e9dad255b112/ijms-20-05760-g001.jpg

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