利用患者来源的诱导多能干细胞对癫痫进行建模和治疗。
Using Patient-Derived Induced Pluripotent Stem Cells to Model and Treat Epilepsies.
作者信息
Du Xixi, Parent Jack M
机构信息
Neuroscience Graduate Program, Medical Scientist Training Program, Department of Neurology, University of Michigan Medical Center, University of Michigan, 5078 BSRB, 109 Zina Pitcher Place, Ann Arbor, MI, 48109-2200, USA,
出版信息
Curr Neurol Neurosci Rep. 2015 Oct;15(10):71. doi: 10.1007/s11910-015-0588-3.
Abstract
Human induced pluripotent stem cells (iPSCs) are transforming the fields of disease modeling and precision therapy. For the treatment of neurological disorders, iPSCs introduce the possibility for targeted cell-based therapies by deriving patient-specific neural tissue in vitro that may ultimately be used for transplantation. We review iPSC technologies and their applications that have already advanced our understanding of neurological disorders, focusing on the epilepsies. We also discuss the application of powerful new tools such as genome editing and multi-well, multi-electrode array recording platforms to iPSC disease modeling and therapy development for the epilepsies. Despite some limitations, the field of iPSCs is evolving rapidly and is quickly becoming vital for understanding mechanisms of genetic epilepsies and for future patient-specific therapeutic applications.
摘要
人类诱导多能干细胞（iPSC）正在改变疾病建模和精准治疗领域。对于神经系统疾病的治疗，iPSC通过在体外衍生患者特异性神经组织为基于细胞的靶向治疗带来了可能性，这些神经组织最终可能用于移植。我们综述了iPSC技术及其应用，这些技术已经增进了我们对神经系统疾病的理解，重点是癫痫。我们还讨论了强大的新工具，如基因组编辑和多孔、多电极阵列记录平台在iPSC癫痫疾病建模和治疗开发中的应用。尽管存在一些局限性，但iPSC领域正在迅速发展，对于理解遗传性癫痫的机制以及未来针对患者的治疗应用正迅速变得至关重要。
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