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Originally published in Science Express on 31 July 2008
Science 29 August 2008: Vol. 321. no. 5893, pp. 1218 - 1221
DOI: 10.1126/science.1158799
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Reports
Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons
John T. Dimos,1*
Kit T. Rodolfa,1,2*
Kathy K. Niakan,1
Laurin M. Weisenthal,1
Hiroshi Mitsumoto,3,4
Wendy Chung,4,5
Gist F. Croft,4,6
Genevieve Saphier,1
Rudy Leibel,5
Robin Goland,7
Hynek Wichterle,4,6
Christopher E. Henderson,4,6
Kevin Eggan1
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.
1 Harvard Stem Cell Institute, Stowers Medical Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.
2 Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
3 Eleanor and Lou Gehrig MDA-ALS Research Center, Neurological Institute, Columbia University Medical Center, New York, NY 10032, USA.
4 Center for Motor Neuron Biology and Disease, Columbia University Medical Center, New York, NY 10032, USA.
5 Division of Molecular Genetics and Naomi Berrie Diabetes Center, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA.
6 Departments of Pathology, Neurology and Neuroscience, Columbia University Medical Center, New York, NY 10032, USA.
7 Department of Medicine and Naomi Berrie Diabetes Center, Columbia University Medical Center, New York, NY 10032, USA.
* These authors contributed equally to this work.
To whom correspondence should be addressed. E-mail: eggan{at}mcb.harvard.edu
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