在培养皿中模拟罕见病发病过程。
Modeling the rare diseases process in dish.
作者信息
Tang Wei
机构信息
National Center for Global Health and Medicine, Tokyo, Japan.
出版信息
Intractable Rare Dis Res. 2018 May;7(2):72. doi: 10.5582/irdr.2018.01048.
Abstract
For rare diseases, how to mimic the pathological progression is of importance for better understanding the molecular mechanism and identifying potential targets. Induced pluripotent stem cells (iPSCs) technology provides an ideal model to in vitro obtain the diseases-associated cells. In this issue of , two birth detect diseases iPS models on phenylketonuria and trisomy 18 were established, and have demonstrated great potential in the research on these rare diseases.
摘要
对于罕见病而言，如何模拟其病理进展对于更好地理解分子机制和识别潜在靶点至关重要。诱导多能干细胞（iPSC）技术为在体外获得疾病相关细胞提供了理想模型。在本期杂志中，建立了苯丙酮尿症和18三体综合征这两种先天性疾病的iPSC模型，并在这些罕见病的研究中展现出了巨大潜力。
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本文引用的文献
1
Establishment of a human trisomy 18 induced pluripotent stem cell line from amniotic fluid cells.从羊水细胞建立人18三体诱导多能干细胞系。
Intractable Rare Dis Res. 2018 May;7(2):94-99. doi: 10.5582/irdr.218.01038.
2
Generation of urine-derived induced pluripotent stem cells from a patient with phenylketonuria.从一名苯丙酮尿症患者中生成尿液来源的诱导多能干细胞。
Intractable Rare Dis Res. 2018 May;7(2):87-93. doi: 10.5582/irdr.2018.01032.
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Modeling rare diseases with induced pluripotent stem cell technology.利用诱导多能干细胞技术建模罕见病。
Mol Cell Probes. 2018 Aug;40:52-59. doi: 10.1016/j.mcp.2018.01.001. Epub 2018 Jan 5.
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Induced pluripotent stem cell technology: a decade of progress.诱导多能干细胞技术：十年进展
Nat Rev Drug Discov. 2017 Feb;16(2):115-130. doi: 10.1038/nrd.2016.245. Epub 2016 Dec 16.
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