Ebert Allison D, Svendsen Clive N
Wisconsin Institutes for Medical Research, 1111 Highland Ave, Room 5033, Madison, WI 53705, USA.
Arch Neurol. 2010 Jun;67(6):665-9. doi: 10.1001/archneurol.2010.89.
Human embryonic stem cells provide a useful source of material for studying basic human development and various disease states. However, ethical issues concerning their procurement limit their acceptance and possible clinical applicability. Recent advances in stem cell technology have provided an alternative source of pluripotent stem cells that does not require the use of an embryo. This review addresses the generation of induced pluripotent stem cells from skin fibroblasts taken from various patient populations, with a specific focus on the pediatric disorder spinal muscular atrophy. These patient-derived cells may help researchers devise more appropriate therapies through a greater understanding of the molecular mechanisms that underlie neuron dysfunction and death in a variety of diseases. Furthermore, they provide an ideal platform for small-molecule screening and subsequent drug development.
人类胚胎干细胞为研究人类基本发育和各种疾病状态提供了有用的材料来源。然而，有关其获取的伦理问题限制了它们被接受的程度以及可能的临床适用性。干细胞技术的最新进展提供了一种无需使用胚胎的多能干细胞替代来源。本综述探讨了从不同患者群体的皮肤成纤维细胞中诱导产生多能干细胞的方法，特别关注小儿疾病脊髓性肌萎缩症。这些源自患者的细胞可能有助于研究人员通过更深入了解多种疾病中神经元功能障碍和死亡背后的分子机制来设计更合适的治疗方法。此外，它们为小分子筛选和后续药物开发提供了理想的平台。
