人iPSC衍生的疾病模型用于药物发现

English | 2023 | ISBN: 3031423488 | 574 Pages | PDF EPUB (True) | 31 MB

Since their development a decade ago, human induced pluripotent stem cells (iPSC) have revolutionized the study of human disease, given rise to regenerative medicine technologies, and provided exceptional opportunities for pharmacologic research. These cells provide an essentially unlimited supply of cell types that are difficult to obtain from patients, such as neurons or cardiomyocytes, or are difficult to maintain in primary cell culture. iPSC can be obtained from patients afflicted with a particular disease but, in combination with recently developed gene editing techniques, can also be modified to generate disease models. Moreover, the new techniques of 3 Dimensional printing and materials science facilitate the generation of organoids that can mirror organs under disease conditions. These properties make iPSC powerful tools to study how diseases develop and how they may be treated. In addition, iPSC can also be used to treat conditions in which the target cell population has been lost and such regenerative approaches hold great promise for currently untreatable diseases, including cardiac failure or photoreceptor degenerations.

自十多年前人类诱导多能干细胞（iPSC）的发展以来，它们已经彻底改变了对人类疾病的研究，催生了再生医学技术，并为药物研究提供了极好的机会。这些细胞提供了几乎无限的类型来源，包括从患者中难以获取的神经元或心肌细胞，或者在原代细胞培养中难以维持的类型。iPSC可以从患有特定疾病的患者中获得，结合最近发展出的基因编辑技术，还可以修改以生成疾病模型。此外，3D打印技术和材料科学的新方法使得可以生成能够反映病理状态下器官的器官oids。这些特性使iPSC成为研究疾病是如何发展的以及如何治疗它们的强大工具。此外，iPSC也可以用于治疗那些目标细胞群体已经丧失的情况，并且再生医学途径对于目前无法治愈的疾病（例如心力衰竭或视网膜退化）具有巨大的潜力。

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