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Using induced pluripotent stem cell neuronal models to study neurodegenerative diseases

机译:使用诱导多能干细胞神经元模型研究神经变性疾病

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Current application of human induced pluripotent stem cells (hiPSCs) technology in patient-specific models of neurodegenerative disorders recapitulate some of key phenotypes of diseases, representing disease-specific cellular modeling and providing a unique platform for therapeutics development. We review recent efforts toward advancing hiPSCs-derived neuronal cell types and highlight their potential use for the development of more complex in vitro models of neurodegenerative diseases by focusing on Alzheimer's disease, Parkinson's disease, Huntington's disease and Amyotrophic lateral sclerosis. We present evidence from previous works on the important phenotypic changes of various neuronal types in these neurological diseases. We also summarize efforts on conducting low- and high-throughput screening experiments with hiPSCs toward developing potential therapeutics for treatment of neurodegenerative diseases. Lastly, we discuss the limitations of hiPSCs culture system in studying neurodegenerative diseases and alternative strategies to overcome these hurdles.
机译:目前人类诱导的多能干细胞(HIPSC)技术在神经变性障碍患者特异性模型中的应用重新涵盖了一些疾病的关键表型,代表了疾病特异性细胞建模,并为治疗发育提供了独特的平台。我们审查了最近努力推进HIPSCS衍生的神经元细胞类型,并通过重点关注阿尔茨海默病,帕廷森病,亨廷顿的疾病和肌萎缩的侧刀死,突出潜在用途,突出了他们对神经变性疾病的更复杂的体外模型的发展。我们提出了先前作品的证据,以对这些神经疾病各种神经元类型的重要表型变化。我们还总结了在开发肝脏展望潜在治疗治疗神经变性疾病的潜在治疗方面的低通量筛选实验的努力。最后,我们探讨了HIPSCS培养系统在研究神经变性疾病和替代策略中的局限,以克服这些障碍。

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