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Modeling Human Neurological and Neurodegenerative Diseases: From Induced Pluripotent Stem Cells to Neuronal Differentiation and Its Applications in Neurotrauma

机译:建模人类神经系统疾病和神经退行性疾病:从诱导多能干细胞到神经元分化及其在神经创伤中的应用

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摘要

With the help of several inducing factors, somatic cells can be reprogrammed to become induced pluripotent stem cell (iPSCs) lines. The success is in obtaining iPSCs almost identical to embryonic stem cells (ESCs), therefore various approaches have been tested and ultimately several ones have succeeded. The importance of these cells is in how they serve as models to unveil the molecular pathways and mechanisms underlying several human diseases, and also in its potential roles in the development of regenerative medicine. They further aid in the development of regenerative medicine, autologous cell therapy and drug or toxicity screening. Here, we provide a comprehensive overview of the recent development in the field of iPSCs research, specifically for modeling human neurological and neurodegenerative diseases, and its applications in neurotrauma. These are mainly characterized by progressive functional or structural neuronal loss rendering them extremely challenging to manage. Many of these diseases, including Parkinson's disease (PD), Huntington's disease (HD), Amyotrophic lateral sclerosis (ALS) and Alzheimer's disease (AD) have been explored in vitro. The main purpose is to generate patient-specific iPS cell lines from the somatic cells that carry mutations or genetic instabilities for the aim of studying their differentiation potential and behavior. This new technology will pave the way for future development in the field of stem cell research anticipating its use in clinical settings and in regenerative medicine in order to treat various human diseases, including neurological and neurodegenerative diseases.
机译:借助几种诱导因子,可以将体细胞重编程为诱导多能干细胞(iPSC)系。成功的原因是获得了与胚胎干细胞(ESC)几乎相同的iPSC,因此已经测试了各种方法,最终有几种方法成功了。这些细胞的重要性在于它们如何充当模型来揭示几种人类疾病的分子途径和机制,以及其在再生医学发展中的潜在作用。它们进一步帮助开发再生医学,自体细胞疗法以及药物或毒性筛选。在这里,我们提供了有关iPSCs研究领域的最新进展的全面概述,特别是用于建模人类神经系统疾病和神经退行性疾病的方法及其在神经创伤中的应用。这些疾病的主要特征是进行性功能或结构神经元的丧失,使其极难处理。已在体外探索了许多这类疾病,包括帕金森氏病(PD),亨廷顿氏病(HD),肌萎缩性侧索硬化症(ALS)和阿尔茨海默氏病(AD)。主要目的是从携带突变或遗传不稳定性的体细胞中生成患者特异性的iPS细胞系,以研究其分化潜能和行为。这项新技术将为干细胞研究领域的未来发展铺平道路,从而有望将其用于临床环境和再生医学中,以治疗各种人类疾病,包括神经系统疾病和神经退行性疾病。

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