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Patient-Derived iPSCs Reprogrammed Into Neurons

In a first, scientists modified patient-derived iPSCs to generate neurons from a person with Amyotrophic Lateral Sclerosis.

Written byAndrea Gawrylewski
| 2 min read

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Researchers have for the first time been able to generate a pluripotent stem cell line from the cells of a patient with a genetic disease, according to a study appearing tomorrow (August 1) in Science.1 The scientists successfully reprogrammed skin cells from an 82-year-old patient with Amyotrophic Lateral Sclerosis (ALS) into healthy motor neurons.

"It's a stunning accomplishment," Neil Cashman, professor of neurology at the University of British Columbia who was not involved in the study, told The Scientist. "That they take somebody at the end of their life with a chronic disease and can still reprogram the cells, it's a slam dunk."

The researchers, led by Kevin Eggan at Harvard University, infected the diseased cells with genetically modified viruses coding for the four transgenes carrying the Yamanaka factors: Klf4, OCT4, SOX2, and c-Myc. This was the same cocktail of genes used by one of two groups last November in ...

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