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Modeling blood diseases with human induced pluripotent stem cells.
Disease Models & Mechanisms ( IF 4.0 ) Pub Date : 2019-06-04 , DOI: 10.1242/dmm.039321
Maria Georgomanoli 1 , Eirini P Papapetrou 2
Affiliation  

Induced pluripotent stem cells (iPSCs) are derived from somatic cells through a reprogramming process, which converts them to a pluripotent state, akin to that of embryonic stem cells. Over the past decade, iPSC models have found increasing applications in the study of human diseases, with blood disorders featuring prominently. Here, we discuss methodological aspects pertaining to iPSC generation, hematopoietic differentiation and gene editing, and provide an overview of uses of iPSCs in modeling the cell and gene therapy of inherited genetic blood disorders, as well as their more recent use as models of myeloid malignancies. We also discuss the strengths and limitations of iPSCs compared to model organisms and other cellular systems commonly used in hematology research.

中文翻译:

用人类诱导的多能干细胞对血液疾病进行建模。

诱导多能干细胞(iPSC)是通过重编程过程从体细胞中获得的,该过程将其转换为类似于胚胎干细胞的多能状态。在过去的十年中,iPSC模型在人类疾病研究中发现了越来越多的应用,其中以血液疾病为主要特征。在这里,我们讨论了与iPSC产生,造血分化和基因编辑有关的方法论方面,并概述了iPSC在建模遗传性遗传性血液疾病的细胞和基因治疗中的用途,以及它们最近作为骨髓恶性肿瘤模型的用途。 。我们还将讨论与模型生物和血液学研究中常用的其他细胞系统相比,iPSC的优势和局限性。
更新日期:2020-08-21
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