Medical Research Institute, Chung-Ang University College of Medicine, Seoul, Korea; Division of Neurology, Department of Medicine, UBC Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
Neuropathology. 2013 Oct;33(5):491-504. doi: 10.1111/neup.12020. Epub 2013 Feb 5.
Human neurodegenerative diseases such as Parkinson's disease (PD), Huntington's disease (HD), amyotrophic lateral sclerosis (ALS) and Alzheimer's disease (AD) are caused by a loss of neurons and glia in the brain or spinal cord. Neurons and glial cells have successfully been generated from stem cells such as embryonic stem cells (ESCs), mesenchymal stem cells (MSCs) and neural stem cells (NSCs), and stem cell-based cell therapies for neurodegenerative diseases have been developed. A recent advance in generation of a new class of pluripotent stem cells, induced pluripotent stem cells (iPSCs), derived from patients' own skin fibroblasts, opens doors for a totally new field of personalized medicine. Transplantation of NSCs, neurons or glia generated from stem cells in animal models of neurodegenerative diseases, including PD, HD, ALS and AD, demonstrates clinical improvement and also life extension of these animals. Additional therapeutic benefits in these animals can be provided by stem cell-mediated gene transfer of therapeutic genes such as neurotrophic factors and enzymes. Although further research is still needed, cell and gene therapy based on stem cells, particularly using neurons and glia derived from iPSCs, ESCs or NSCs, will become a routine treatment for patients suffering from neurodegenerative diseases and also stroke and spinal cord injury.
人类神经退行性疾病，如帕金森病 (PD)、亨廷顿病 (HD)、肌萎缩侧索硬化症 (ALS) 和阿尔茨海默病 (AD)，是由大脑或脊髓中的神经元和神经胶质细胞丧失引起的。已经成功地从胚胎干细胞 (ESCs)、间充质干细胞 (MSCs) 和神经干细胞 (NSCs) 等干细胞中生成神经元和神经胶质细胞，并且已经开发出基于干细胞的神经退行性疾病细胞疗法。最近在生成一类新的多能干细胞，即诱导多能干细胞 (iPSCs) 方面的进展，这些细胞源自患者自身的皮肤成纤维细胞，为个性化医学的全新领域开辟了道路。将 NSCs、神经元或神经胶质细胞从 PD、HD、ALS 和 AD 等神经退行性疾病动物模型中的干细胞中移植，可改善这些动物的临床症状，并延长其寿命。通过干细胞介导的神经营养因子和酶等治疗基因的转移，还可以为这些动物提供额外的治疗益处。尽管还需要进一步的研究，但基于干细胞的细胞和基因疗法，特别是使用源自 iPSCs、ESCs 或 NSCs 的神经元和神经胶质细胞，将成为治疗神经退行性疾病以及中风和脊髓损伤患者的常规治疗方法。
