Papapetrou Eirini P, Sadelain Michel
Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan-Kettering Cancer Center 1275 York Avenue, New York, NY 10065, USA.
F1000 Med Rep. 2010 Jun 16;2:44. doi: 10.3410/M2-44.
The direct reprogramming of human somatic cells to induced pluripotent stem cells (iPSCs) offers exciting prospects for disease modelling and regenerative medicine. Several recent reports support the feasibility of generating various blood cell types from iPSCs through in vitro-directed differentiation. However, the derivation of hematopoietic stem cells (HSCs) capable of long-term reconstitution of all hematopoietic lineages appears to be more challenging. These hurdles notwithstanding, cell engineering strategies aiming to correct genetic defects at the stem cell level are already emerging. Robust methodologies for the generation of definitive human HSCs conferring high-level, multilineage, long-term, hematopoietic reconstitution thus are direly needed before the therapeutic potential and safety of iPSC-derived cell products can be thoroughly investigated.
将人类体细胞直接重编程为诱导多能干细胞(iPSC)为疾病建模和再生医学带来了令人兴奋的前景。最近的几份报告支持了通过体外定向分化从iPSC生成各种血细胞类型的可行性。然而,能够长期重建所有造血谱系的造血干细胞(HSC)的衍生似乎更具挑战性。尽管存在这些障碍,旨在在干细胞水平纠正遗传缺陷的细胞工程策略已经出现。因此,在能够彻底研究iPSC衍生细胞产品的治疗潜力和安全性之前,迫切需要强大的方法来生成能够进行高水平、多谱系、长期造血重建的确定性人类HSC。