Induced pluripotent stem cells for modelling human diseases

被引:62
作者
Unternaehrer, Juli J. [1 ,4 ,5 ]
Daley, George Q. [1 ,2 ,3 ,4 ,5 ,6 ]
机构
[1] Childrens Hosp, Div Pediat Hematol Oncol, Stem Cell Transplantat Program, Boston, MA 02115 USA
[2] Childrens Hosp, Howard Hughes Med Inst, Manton Ctr Orphan Dis Res, Boston, MA 02115 USA
[3] Dana Farber Canc Inst, Boston, MA 02115 USA
[4] Brigham & Womens Hosp, Div Hematol, Boston, MA 02115 USA
[5] Harvard Univ, Sch Med, Dept Biol Chem & Mol Pharmacol, Boston, MA 02115 USA
[6] Harvard Stem Cell Inst, Boston, MA 02115 USA
关键词
induced pluripotent stem cells; disease modelling; reprogramming; embryonic stem cells; FRAGILE-X-SYNDROME; LONG-QT SYNDROME; DIRECTED DIFFERENTIATION; PARKINSONS-DISEASE; MOTOR-NEURONS; PATIENT; GENERATION; ANEMIA; PATHOGENESIS; FIBROBLASTS;
D O I
10.1098/rstb.2011.0017
中图分类号
Q [生物科学];
学科分类号
07 ; 0710 ; 09 ;
摘要
Research into the pathophysiological mechanisms of human disease and the development of targeted therapies have been hindered by a lack of predictive disease models that can be experimentally manipulated in vitro. This review describes the current state of modelling human diseases with the use of human induced pluripotent stem (iPS) cell lines. To date, a variety of neurodegenerative diseases, haematopoietic disorders, metabolic conditions and cardiovascular pathologies have been captured in a Petri dish through reprogramming of patient cells into iPS cells followed by directed differentiation of disease-relevant cells and tissues. However, realizing the true promise of iPS cells for advancing our basic understanding of disease and ultimately providing novel cell-based therapies will require more refined protocols for generating the highly specialized cells affected by disease, coupled with strategies for drug discovery and cell transplantation.
引用
收藏
页码:2274 / 2285
页数:12
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