Nature：定制iPS细胞（图）

　　

生物通报道，来自西班牙巴萨罗那再生医学中心，美国Salk研究所等处的研究人员在最近的一期Nature上发表iPS研究进展，Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells。

 

范可尼贫血(Fanconi anemia）是一种罕见的常染色体隐性遗传性血液系统疾病，属于先天性再障，称为Fanconi贫血(范可尼贫血)，这类病人除有典型再障表现外，还伴有多发性的先天畸形(皮肤棕色色素沉着，骨骼畸形、性发育不全等)。其病因可能是染色体的异常。同时，还会伴有精子减少等其它特征。经常与其它疾病伴发，因此科学家一直在探索这个疾病的根本病因。

 

本期Nature上Juan Carlos Izpisúa Belmonte带领的研究小组将范可尼贫血患者的造血细胞用iPS诱导技术成功诱导成iPS细胞，再经定向诱导将其分化成健康的造血干细胞样细胞，它们能产生属于骨髓细胞系和类红细胞系的不含疾病的造血祖细胞。

 

图片说明：诱导的范可尼贫血iPS细胞

 

Juan Carlos Izpisúa Belmonte等人希望这种特异的iPS细胞不仅能作为疾病研究模型还能成为范可尼贫血的特定治疗方案。

（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

Ángel Raya1,2,3, Ignasi Rodríguez-Pizà1, Guillermo Guenechea4,5, Rita Vassena1, Susana Navarro4,5, María José Barrero1, Antonella Consiglio1,6, Maria Castellà5,7, Paula Río4,5, Eduard Sleep1,3, Federico González1, Gustavo Tiscornia1, Elena Garreta1,3, Trond Aasen1,3, Anna Veiga1, Inder M. Verma8, Jordi Surrallés5,7, Juan Bueren4,5 & Juan Carlos Izpisúa Belmonte1,9

 

Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain

Institució Catalana de Recerca i Estudis Avançats (ICREA),

Networking Center of Biomedical Research in Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN),

Hematopoiesis and Gene Therapy Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Av. Complutense 22, 28040 Madrid, Spain

Networking Center of Biomedical Research in Rare Diseases (CIBERER),

Department of Biomedical Science and Biotechnology, University of Brescia, Viale Europa 11, 25123 Brescia, Italy

Department of Genetics and Microbiology, Universitat Autonoma de Barcelona, 08193 Bellaterra, Spain

Laboratory of Genetics,

Gene Expression Laboratory, Salk Institute for Biological Studies, 10010 North Torrey Pines Road, La Jolla, California 92037, USA

Correspondence to: Juan Carlos Izpisúa Belmonte1,9 Correspondence and requests for materials should be addressed to J.C.I.B. (Email: [e-mail] or Email: [e-mail]).

 

【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.