Department of Molecular Cell Biology, Regenerative Medicine Program, Leiden University Medical Center, Leiden, the Netherlands.
Ann N Y Acad Sci. 2012 Aug;1266:179-88. doi: 10.1111/j.1749-6632.2012.06629.x.
Many life-threatening hematological diseases are now treated by bone marrow transplantations, i.e., infusion of hematopoietic stem cells (HSCs). HSC transplantations are a valid option for the treatment of a variety of metabolic disorders, and even for solid tumors and some refractory severe autoimmune diseases. Unfortunately, the frequency and outcome of HSC transplantations are limited by a shortage of suitable donors. Induced pluripotent stem cells (iPSCs)--somatic cells that have acquired pluripotent stem cell characteristics by the ectopic expression of pluripotency-inducing factors--have been proposed as an alternative source of HSCs. Possible applications include cells of autologous, of autologous and genetically modified, or of allogeneic origin. Here, we provide a perspective on the distinct opportunities of iPSCs and discuss the challenges that lie ahead.
现在，许多危及生命的血液系统疾病都可以通过骨髓移植（即输注造血干细胞，HSCs）来治疗。HSC 移植是治疗多种代谢紊乱的有效选择，甚至对实体瘤和一些难治性严重自身免疫性疾病也是如此。不幸的是，由于合适供体的缺乏，HSC 移植的频率和结果受到限制。诱导多能干细胞（iPSCs）——通过异位表达多能性诱导因子获得多能性干细胞特征的体细胞——已被提议作为 HSCs 的替代来源。可能的应用包括自体、自体和基因修饰的或同种异体来源的细胞。在这里，我们提供了对 iPSCs 的不同机会的看法，并讨论了未来的挑战。
