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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,1Laurin M. Weisenthal,1Hiroshi Mitsumoto,3,4Wendy Chung,4,5Gist F. Croft,4,6Genevieve Saphier,1Rudy Leibel,5Robin Goland,7Hynek Wichterle,4,6Christopher E. Henderson,4,6Kevin Eggan1
The generation of pluripotent stem cells from an individualpatient would enable the large-scale production of the celltypes affected by that patient's disease. These cells couldin turn be used for disease modeling, drug discovery, and eventuallyautologous cell replacement therapies. Although recent studieshave demonstrated the reprogramming of human fibroblasts toa pluripotent state, it remains unclear whether these inducedpluripotent stem (iPS) cells can be produced directly from elderlypatients with chronic disease. We have generated iPS cells froman 82-year-old woman diagnosed with a familial form of amyotrophiclateral sclerosis (ALS). These patient-specific iPS cells possessproperties of embryonic stem cells and were successfully directedto differentiate into motor neurons, the cell type destroyedin 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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[DOI: 10.1126/science.1163475] |Summary »|Full Text »|PDF »
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