Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons.
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.
Dimos
John T
JT
Harvard Stem Cell Institute, Stowers Medical Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.
Rodolfa
Kit T
KT
Niakan
Kathy K
KK
Weisenthal
Laurin M
LM
Mitsumoto
Hiroshi
H
Chung
Wendy
W
Croft
Gist F
GF
Saphier
Genevieve
G
Leibel
Rudy
R
Goland
Robin
R
Wichterle
Hynek
H
Henderson
Christopher E
CE
Eggan
Kevin
K
D000369:Aged, 80 and over; D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics / Q000473:pathology* / Q000503:physiopathology; D002454:Cell Differentiation; D002460:Cell Line; D065150:Cellular Reprogramming; D053595:Embryonic Stem Cells / Q000166:cytology; D005260:Female; D005347:Fibroblasts / Q000166:cytology*; D015870:Gene Expression; D006801:Humans; D009046:Motor Neurons / Q000166:cytology* / Q000378:metabolism; D009457:Neuroglia / Q000166:cytology; D039904:Pluripotent Stem Cells / Q000166:cytology*; D012190:Retroviridae / Q000235:genetics; D013116:Spinal Cord / Q000166:cytology; D013482:Superoxide Dismutase / Q000235:genetics / Q000378:metabolism; D000072105:Superoxide Dismutase-1; D014157:Transcription Factors / Q000235:genetics / Q000378:metabolism; D014161:Transduction, Genetic
C000606290:SOD1 protein, human; D014157:Transcription Factors; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1