Centre of Regenerative Medicine in Barcelona, Barcelona, Spain.
Nat Med. 2011 Dec;17(12):1570-6. doi: 10.1038/nm.2504.
The derivation of induced pluripotent cells (iPSCs) from individuals suffering from genetic syndromes offers new opportunities for basic research into these diseases and the development of therapeutic compounds. iPSCs can self renew and can be differentiated to many cell types, offering a potentially unlimited source of material for study. In this review we discuss the conceptual and practical issues to consider when attempting to model genetic diseases using iPSCs.
诱导多能干细胞(iPSCs)来源于患有遗传综合征的个体,为这些疾病的基础研究和治疗性化合物的开发提供了新的机会。iPSCs 可以自我更新,并且可以分化为多种细胞类型,为研究提供了潜在的无限材料来源。在这篇综述中,我们讨论了使用 iPSCs 模拟遗传疾病时需要考虑的概念和实际问题。