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通过 IPS 的方式对范可尼贫血的造血前体细胞的基因修正
诱导性多能干细胞的产生是形成病人特异性的多能细胞和为建立人类疾病模型提供了宝贵的实验平台。病人特异性 iPS 细胞也被认为具有伟大的治疗潜能，虽然还没有直接证据。在此我们发现, 在修正的遗传缺陷方面，从范可尼贫血患者体细胞可以重新编程，产生的病人特异性 iPS 细胞。这些细胞系与人类健康个体胚胎干细胞和 iPS 细胞系没有多大差别。重要的是, 我们的分析表明, 纠正了范可尼贫血 特异性 iPS 细胞能增加正常的髓细胞和红细胞的血液祖细胞比例，这样细胞是不带有疾病的。这些数据提供足够的证据可以说明使用 ips 细胞技术的能够产生基因修正的病人特异性细胞，这些细胞具有潜在的临床治疗价值。
Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells
The generation of induced pluripotent stem (iPS) cells has enabled the derivation of patient-specific pluripotent cells and provided valuable experimental platforms to model human disease. Patient-specific iPS cells are also thought to hold great therapeutic potential, although direct evidence for this is still lacking. Here we show that, on correction of the genetic defect,somatic cells from Fanconi anaemia patients can be reprogrammed to pluripotency to generate patient-specific iPS cells. These cell lines appear indistinguishable from human embryonic stem cells and iPS cells from healthy individuals. Most importantly, we show that corrected Fanconi-anaemia-specific iPS cells can give rise to haematopoietic progenitors of the myeloid and erythroid lineages that are phenotypically normal, that is, disease-free. These data offer proof-of-concept that iPS cell technology can be used for the generation of disease-corrected, patient-specific cells with potential value for cell therapy applications.
