神经退行性疾病和耐药性癫痫的干细胞和基因治疗的最新进展。
Recent advancements in stem cell and gene therapies for neurological disorders and intractable epilepsy.
机构信息
Department of Biology and Program in Neuroscience and Behavior, Hall Atwater Laboratory, 52 Lawn Avenue, Wesleyan University, Middletown, CT 06459, USA.
出版信息
Neuropharmacology. 2010 May;58(6):855-64. doi: 10.1016/j.neuropharm.2010.01.019. Epub 2010 Feb 8.
Abstract
The potential applications of stem cell therapies for treating neurological disorders are enormous. Many laboratories are focusing on stem cell treatments for CNS diseases, including spinal cord injury, Amyotrophic lateral sclerosis, Parkinson's disease, Huntington's disease, multiple sclerosis, stroke, traumatic brain injury, and epilepsy. Among the many stem cell types under testing for neurological treatments, the most common are fetal and adult brain stem cells, embryonic stem cells, induced pluripotent stem cells, and mesenchymal stem cells. An expanding toolbox of molecular probes is now available to allow analyses of neural stem cell fates prior to and after transplantation. Concomitantly, protocols are being developed to direct the fates of stem cell-derived neural progenitors, and also to screen stem cells for tumorigenicity and aneuploidy. The rapid progress in the field suggests that novel stem cell and gene therapies for neurological disorders are in the pipeline.
摘要
干细胞疗法在治疗神经紊乱方面的潜在应用是巨大的。许多实验室都专注于针对中枢神经系统疾病的干细胞治疗，包括脊髓损伤、肌萎缩性侧索硬化症、帕金森病、亨廷顿病、多发性硬化症、中风、创伤性脑损伤和癫痫。在为神经治疗测试的众多干细胞类型中，最常见的是胎儿和成人脑干细胞、胚胎干细胞、诱导多能干细胞和间充质干细胞。现在有一个不断扩大的分子探针工具箱，可用于分析移植前后神经干细胞的命运。同时，正在制定方案来指导干细胞衍生的神经祖细胞的命运，还筛选用于肿瘤形成和非整倍性的干细胞。该领域的快速进展表明，针对神经紊乱的新型干细胞和基因疗法正在开发中。
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