Available Technology
Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons
Technology:
iPS cell lines generated from patients suffering from familial or sporadic forms of ALS
Markets Addressed
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder in which motor neuron loss in the spinal cord and motor cortex leads to progressive paralysis and death. Approximately 5,600 people in the U.S. are diagnosed with ALS each year. The incidence of ALS is two per 100,000 people, and it is estimated that as many as 30,000 Americans may have the disease at any given time. Studies aimed at understanding the root causes of motor neuron death in ALS and efforts to develop new therapeutics would be greatly advanced if a robust supply of human motor neurons carrying the genes responsible for this condition could be generated. In addition, a long sought goal of regenerative and transplantation medicine has been to use cells or tissues genetically matched to a patient.
Innovations and Advantages
Kevin Eggan’s group of Harvard University has successfully generated induced pluripotent stem cells (iPS cells) directly from patients. A number of iPS cell lines were generated using fibroblasts taken directly from patients suffering from familial or sporadic forms of ALS. These iPS cells are truly pluripotent stem cell lines with a gene expression signature similar to human embryonic stem cells and are capable of differentiating into cell types derived from the three embryonic germ layers. These cells are immortal and can be grown in the laboratory indefinitely, and can also generate any of the body’s specialized cell types. Most importantly, these iPS cells can be robustly differentiated into motor neurons, the cell type destroyed in ALS.
The patient-specific iPS cells produced from familial ALS patients will be important tools for further studies of mechanisms by which familial disease arises. Also, since more than 90% of ALS patients are afflicted by a sporadic form of disease, patient-specific iPS cells generated from individuals with sporadic disease would carry the precise constellation of genetic information associated with pathology in that person. This approach would allow study of living motor neurons generated from ALS cases with unknown genetic lesions, providing insight into their intrinsic survival properties, their interactions with other cell types, and their susceptibility to the environmental conditions that are considered to play an important role in ALS pathogenesis.
Additional Information
Intellectual Property Status: Patent pending.
Publication:
Boulting GL, Kiskinis E, Croft GF, Amoroso MW, Oakley DH, Wainger BJ, Williams DJ, Kahler DJ, Yamaki M, Davidow L, Rodolfa CT, Dimos JT, Mikkilineni S, Macdermott AB, Woolf CJ, Henderson CE, Wichterle H, Eggan K. 2011. A functionally characterized test set of human induced pluripotent stem cells. Nat Biotechnol. 29(3):279-86.
John T. Dimos, Kit T. Rodolfa, Kathy K. Niakan, Laurin M. Weisenthal, Hiroshi Mitsumoto, Wendy Chung, Gist F. Croft, Genevieve Saphier, Rudy Leibel, Robin Goland, Hynek Wichterle, Christopher E. Henderson, and Kevin Eggan. 2008. Science 321 (5893), 1218.
See article in the Harvard Gazette.
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Inventor(s):
Eggan, Kevin Carl
Categories:
For further information, please contact:
Vivian Berlin, Director of Business Development
(617) 496-0474
Reference Harvard Case #3205