Division of Pediatric Hematology Oncology, Children's Hospital, Boston, Massachusetts 02115, USA.
Cytotherapy. 2009;11(8):980-9. doi: 10.3109/14653240903348319.
Induced pluripotent stem cells (iPSC) were first generated from somatic cells via the transduction of four 'Yamanaka' factors, Oct4, Sox2, Klf4 and c-Myc. Because iPSC are similar to embryonic stem cells (ESC) and can be differentiated into any cell type of choice, iPSC have the potential to become a platform for personalized medicine by allowing a patient's own cells to become a source of therapeutic tissue. This review describes the main challenges in iPSC technology by focusing on its application to hematologic diseases. The explosive interest in improving iPSC technology has generated numerous genetic and chemical methods for iPSC derivation, but these methods must be evaluated comparatively for their safety and efficacy because there are risks of genetic abnormalities and oncogenesis. Competent iPSC will need to be selected carefully based on physical, genetic and functional criteria, and differentiated efficiently into hematopoietic stem cells via modulation of several signaling pathways before they prove valuable in the clinic.
诱导多能干细胞(iPSC)最初是通过转导四种“山中因子”(Oct4、Sox2、Klf4 和 c-Myc)从体细胞中产生的。由于 iPSC 类似于胚胎干细胞(ESC),并且可以分化为任何所需的细胞类型,因此 iPSC 有可能通过允许患者自身的细胞成为治疗性组织的来源,成为个性化医疗的平台。本综述通过关注其在血液疾病中的应用,描述了 iPSC 技术的主要挑战。对改善 iPSC 技术的巨大兴趣产生了许多用于 iPSC 衍生的遗传和化学方法,但这些方法必须进行安全性和有效性的比较评估,因为存在遗传异常和致癌的风险。在临床应用中具有价值之前,需要根据物理、遗传和功能标准仔细选择有能力的 iPSC,并通过调节几种信号通路有效地分化为造血干细胞。