使用诱导多能干细胞神经元模型来研究神经退行性疾病。
Using induced pluripotent stem cell neuronal models to study neurodegenerative diseases.
机构信息
Center of Implant Dentistry, School of Stomatology, China Medical University, Shenyang 110002, China; Department of Physiology & Biophysics, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.
Department of Physiology & Biophysics, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.
出版信息
Biochim Biophys Acta Mol Basis Dis. 2020 Apr 1;1866(4):165431. doi: 10.1016/j.bbadis.2019.03.004. Epub 2019 Mar 18.
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.
当前，人类诱导多能干细胞（hiPSC）技术在神经退行性疾病的患者特异性模型中的应用再现了疾病的一些关键表型，代表了疾病特异性细胞建模，并为治疗药物的开发提供了独特的平台。我们综述了在推进 hiPSC 衍生神经元细胞类型方面的最新进展，并通过关注阿尔茨海默病、帕金森病、亨廷顿病和肌萎缩性侧索硬化症，强调了它们在开发更复杂的神经退行性疾病体外模型方面的潜在用途。我们展示了之前关于这些神经疾病中各种神经元类型的重要表型变化的研究证据。我们还总结了使用 hiPSC 进行高通量和低通量筛选实验以开发神经退行性疾病治疗方法的努力。最后，我们讨论了 hiPSC 培养系统在研究神经退行性疾病中的局限性以及克服这些障碍的替代策略。
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