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神经退行性疾病,包括帕金森病、阿尔茨海默病和肌萎缩侧索硬化症等的共同特征是在中枢神经系统的不同部位发生特发性神经元丢失。这些神经元的丢失给病人造成了一系列相应的功能障碍。应用人类胚胎干细胞进行细胞替代治疗曾引起人们很大的兴趣,但是一些伦理学问题阻碍了该研究的发展。通过导入特定的转录因子,体细胞能够被诱导为具有胚胎干细胞特性的细胞,即诱导多能干细胞(induced pluripotent stem cells, iPS cells)。获取人类iPS细胞并不涉及明显的伦理问题,并且运用病人特异性的iPS细胞能使自体移植成为可能。因此,iPS细胞有可能成为细胞替代治疗中可靠的细胞来源。此外,利用iPS细胞,人们还能在体外直接研究病变神经细胞的表型以及神经细胞在特定致病因子作用下的疾病易感性,有助于揭示神经退行性疾病的内在机制。本文综述了iPS细胞用于神经退行性疾病细胞治疗的最新进展,并探讨了其在建立疾病的细胞模型中的潜在价值。
A common feature of neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease and amyotrophic lateral sclerosis, is the loss of idiopathic neurons in different parts of the central nervous system. The loss of these neurons causes a series of corresponding dysfunctions to the patient. The use of human embryonic stem cells for cell replacement therapy has aroused great interest, but some ethical problems hinder the development of the study. By introducing specific transcription factors, somatic cells can be induced into cells that have the characteristics of embryonic stem cells, that is, induced pluripotent stem cells (iPS cells). Obtaining human iPS cells does not involve significant ethical issues and autologous transplantation is possible using patient-specific iPS cells. Therefore, iPS cells may become a reliable source of cells in cell replacement therapy. In addition, using iPS cells, it is also possible to directly study the phenotype of diseased nerve cells and the susceptibility of nerve cells to disease under the action of specific virulence factors in vitro, so as to reveal the underlying mechanism of neurodegenerative diseases. This review summarizes the recent advances in the use of iPS cells in the treatment of neurodegenerative diseases and explores its potential value in establishing cellular models of disease.