Modeling Human Neurological and Neurodegenerative Diseases: From Induced Pluripotent Stem Cells to Neuronal Differentiation and Its Applications in Neurotrauma.
Modeling Human Neurological and Neurodegenerative Diseases: From Induced Pluripotent Stem Cells to Neuronal Differentiation and Its Applications in Neurotrauma.
复制标题
模拟人类神经和神经退行性疾病：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。
DOI：
10.3389/fnmol.2017.00050
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发表时间：
2017
影响因子：
4.8
通讯作者：
Abou-Kheir W
中科院分区：
文献类型：
--
作者：
Bahmad H;Hadadeh O;Chamaa F;Cheaito K;Darwish B;Makkawi AK;Abou-Kheir W
在几种诱导因子的帮助下，体细胞可以被重新编程为诱导多能干细胞(IPSCs)系。成功的是获得了几乎与胚胎干细胞(ESCs)完全相同的IPSCs，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作为模型来揭示几种人类疾病背后的分子途径和机制，以及它们在再生医学发展中的潜在作用。它们还有助于再生医学、自体细胞疗法和药物或毒性筛选的发展。在这里，我们全面概述了IPSCs研究领域的最新进展，特别是在模拟人类神经和神经退行性疾病方面，以及它在神经创伤中的应用。这些疾病的主要特征是进行性的功能性或结构性神经元丢失，这使得它们的管理具有极大的挑战性。其中许多疾病，包括帕金森氏病(PD)、亨廷顿病(HD)、肌萎缩侧索硬化症(ALS)和阿尔茨海默病(AD)，已经在体外进行了研究。主要目的是从携带突变或遗传不稳定性的体细胞中产生患者特有的iPS细胞系，以研究其分化潜力和行为。这项新技术将为干细胞研究领域的未来发展铺平道路，预计将在临床环境和再生医学中使用，以治疗各种人类疾病，包括神经和神经退行性疾病。
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.