Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons

被引:1412
作者
Dimos, John T. [1 ]
Rodolfa, Kit T. [1 ,2 ]
Niakan, Kathy K. [1 ]
Weisenthal, Laurin M. [1 ]
Mitsumoto, Hiroshi [3 ,4 ]
Chung, Wendy [4 ,5 ,6 ]
Croft, Gist F. [4 ,7 ,8 ]
Saphier, Genevieve [1 ]
Leibel, Rudy [5 ,6 ]
Goland, Robin [9 ,10 ]
Wichterle, Hynek [4 ,7 ,8 ]
Henderson, Christopher E. [4 ,7 ,8 ]
Eggan, Kevin [1 ]
机构
[1] Harvard Univ, Harvard Stem Cell Inst, Stowers Med Inst, Dept Stem Cell & Regenerat Biol, Cambridge, MA 02138 USA
[2] Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
[3] Columbia Univ, Med Ctr, Inst Neurol, Eleanor & Lou Gehrig MDA ALS Res Ctr, New York, NY 10032 USA
[4] Columbia Univ, Med Ctr, Ctr Motor Neuron Biol & Dis, New York, NY 10032 USA
[5] Columbia Univ, Coll Phys & Surg, Div Mol Genet, New York, NY 10032 USA
[6] Columbia Univ, Coll Phys & Surg, Naomi Berrie Diabet Ctr, New York, NY 10032 USA
[7] Columbia Univ, Med Ctr, Dept Pathol, New York, NY 10032 USA
[8] Columbia Univ, Med Ctr, Dept Neurol & Neurosci, New York, NY 10032 USA
[9] Columbia Univ, Med Ctr, Dept Med, New York, NY 10032 USA
[10] Columbia Univ, Med Ctr, Naomi Berrie Diabet Ctr, New York, NY 10032 USA
关键词
D O I
10.1126/science.1158799
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
The generation of pluripotent stem cells from an individual patient would enable the large- scale production of the cell types affected by that patient's disease. These cells could in turn be used for disease modeling, drug discovery, and eventually autologous cell replacement therapies. Although recent studies have demonstrated the reprogramming of human fibroblasts to a pluripotent state, it remains unclear whether these induced pluripotent stem (iPS) cells can be produced directly from elderly patients with chronic disease. We have generated iPS cells from an 82- year- old woman diagnosed with a familial form of amyotrophic lateral sclerosis (ALS). These patient- specific iPS cells possess properties of embryonic stem cells and were successfully directed to differentiate into motor neurons, the cell type destroyed in ALS.
引用
收藏
页码:1218 / 1221
页数:4
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