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Induced Pluripotent Stem Cells Derived from Alzheimer’s Disease Patients: The Promise the Hope and the Path Ahead

机译:阿尔茨海默氏病患者衍生的多能干细胞:承诺希望和未来之路

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摘要

The future hope of generated induced pluripotent stem cells (iPS cells) from Alzheimer’s disease patients is multifold. Firstly, they may help to uncover novel mechanisms of the disease, which could lead to the development of new and unprecedented drugs for patients and secondly, they could also be directly used for screening and testing of potential new compounds for drug discovery. In addition, in the case of familial known mutations, these cells could be targeted by use of advanced gene-editing techniques to correct the mutation and be used for future cell transplantation therapies. This review summarizes the work so far in regards to production and characterization of iPS cell lines from both sporadic and familial Alzheimer’s patients and from other iPS cell lines that may help to model the disease. It provides a detailed comparison between published reports and states the present hurdles we face with this new technology. The promise of new gene-editing techniques and accelerated aging models also aim to move this field further by providing better control cell lines for comparisons and potentially better phenotypes, respectively.
机译:从阿尔茨海默氏病患者中产生诱导性多能干细胞(iPS细胞)的未来希望是多重的。首先,它们可能有助于发现该疾病的新机制,从而可能导致为患者开发出前所未有的新药物;其次,它们还可以直接用于筛选和测试潜在的新化合物以发现新药。此外,在家族性已知突变的情况下,可以通过使用先进的基因编辑技术来靶向这些细胞以纠正突变,并将其用于未来的细胞移植治疗。这篇综述总结了迄今为止针对散发性和家族性阿尔茨海默氏病患者以及可能有助于对该疾病建模的其他iPS细胞系生产和表征iPS细胞系所做的工作。它提供了已发布报告之间的详细比较,并阐明了使用该新技术目前面临的障碍。新的基因编辑技术和加速衰老模型的前景也旨在通过分别提供更好的对照细胞系进行比较和潜在更好的表型来进一步推动这一领域的发展。

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