Nature：个性化iPS干细胞（图）

　　

生物通报道，Nature杂志在线发表了来自西班牙巴萨罗那再生医学中心，意大利，美国的研究者最新的干细胞研究文章，Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells。

 

近期Cell Stem Cell发表了两篇文章，为iPS技术走向临床奠定了基础，一篇是来自斯克里普斯研究所（Scripps Research Institute）的丁盛（Sheng Ding，生物通译），他用重组的蛋白就能诱导出多能干细胞。丁盛博士的这一成果在小鼠模型上得以验证。时隔不久，哈佛大学的韩裔教授Kwang-Soo Kim小组成功地在人成纤维细胞中验证这一技术，文章也刊登在《Cell Stem Cell》上。（生物通前期相关报道文章：

http://www.ebiotrade.com/newsf/2009-5/2009531171753355.htm  http://www.ebiotrade.com/newsf/2009-4/2009424171426408.htm）

 

这两项技术的突破无疑拉近了iPS走向临床的距离。而Nature的这篇文章为定制个性化的iPS治疗技术奠定了基础。

 

用病人自身的细胞诱导iPS细胞用于治疗可有效避免患者的免疫排斥问题。尽管科学界一直认同这一观点，但是一直缺乏实证证实。在本研究中，研究小组用基因疗法改造范可尼贫血症患者异常的细胞（Fanconi贫血是一种罕见的隐性遗传性疾病,临床常以先天性畸形、进行性骨髓衰竭和遗传性肿瘤倾向为主要表现而确诊），之后用iPS技术诱导这些改造后的正常细胞，将其诱导成胚胎干细胞样细胞，这些细胞与胚胎干细胞和正常人的iPS细胞没有差别。

染色后的Fanconi贫血患者改造iPS

Genetically-corrected fibroblasts (above)

are reprogrammed to give rise to

disease-free blood cell precursors (below)

 

在经过定向分化诱导这些细胞分化为正常的造血祖细胞，这些造血祖细胞表型正常。研究者认为，这些研究数据对iPS细胞在遗传疾病治疗具有指导意义，一旦时机成熟，研究者就能将这些成果转化为现实。

 

（生物通 小茜）

 

生物通推荐原文检索：Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

【Abstract】

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.