Patient-specific pluripotent stem cells in neurological diseases.
Patient-specific pluripotent stem cells in neurological diseases.
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DOI：
10.4061/2011/212487
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发表时间：
2011
影响因子：
4.3
通讯作者：
Genc K
中科院分区：
文献类型：
--
作者：
Durnaoglu S;Genc S;Genc K
许多人类神经系统疾病目前无法治愈，并导致毁灭性的神经后遗症。来源于成人体细胞的诱导多能干细胞（iPSC）的日益增多为细胞替代策略和广泛的人类神经系统疾病的疾病相关基础研究提供了新的前景。基于患者特异性iPSC的神经遗传性和神经退行性疾病建模是一种新兴的有效工具，用于体外建模以了解疾病并筛选改变疾病过程的基因和药物。近年来，随着iPSC研究的指数增长，人类iPSC已成功地用不同的技术从各种细胞类型中衍生出来。尽管仍有很多关于患者特异性iPSC安全性、重编程机制、指导特定重编程的更好方法、细胞移植物的理想细胞来源以及移植干细胞导致增强的功能恢复和结构重组的机制的研究，但iPSC治疗潜力的发现为治疗不可治愈的神经系统疾病提供了新的机会。然而，基于iPSC的治疗策略需要在神经系统疾病的临床前动物模型中进行彻底评估，然后才能应用于临床环境。
Many human neurological diseases are not currently curable and result in devastating neurologic sequelae. The increasing availability of induced pluripotent stem cells (iPSCs) derived from adult human somatic cells provides new prospects for cellreplacement strategies and disease-related basic research in a broad spectrum of human neurologic diseases. Patient-specific iPSC-based modeling of neurogenetic and neurodegenerative diseases is an emerging efficient tool for in vitro modeling to understand disease and to screen for genes and drugs that modify the disease process. With the exponential increase in iPSC research in recent years, human iPSCs have been successfully derived with different technologies and from various cell types. Although there remain a great deal to learn about patient-specific iPSC safety, the reprogramming mechanisms, better ways to direct a specific reprogramming, ideal cell source for cellular grafts, and the mechanisms by which transplanted stem cells lead to an enhanced functional recovery and structural reorganization, the discovery of the therapeutic potential of iPSCs offers new opportunities for the treatment of incurable neurologic diseases. However, iPSC-based therapeutic strategies need to be thoroughly evaluated in preclinical animal models of neurological diseases before they can be applied in a clinical setting.
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