Feat could one day lead to tailor-made cells to treat fatal disease, researchers say
THURSDAY, July 31 (HealthDay News) -- Scientists have turned skin cells from patients with Lou Gehrig's disease into motor neurons that are genetically identical to the patients' own neurons.
An unlimited number of these neurons can now be created and studied in the laboratory, a capability which should result in a better understanding of the disease and, one day, lead to new treatments or even the production of healthy cells that can replace the diseased ones.
"The hope of some scientists is that they might be able to harness stem cells and program them to generate pluripotent stem cell lines [capable of differentiating into many different types of cells] which have the genes of patients," said Kevin Eggan, co-author of a paper appearing July 31 in the online version of Science. "This would open up the possibility of producing a large supply of immune-matched cells to that patient that could be used in transplantation methodologies."
"The other hope, and one that's much closer upon us . . . is if you could produce the cell types that become sick in that person, you might be able to use them in the laboratory to come to understand basic aspects of the disease and take the study of disease out of patients, where it's very difficult, and put it into the Petri dish," added Eggan, who is a principal faculty member at the Harvard Stem Cell Institute and spoke about the research at a teleconference Wednesday.
However, the actual therapeutic potential of this approach is still years away.
Lou Gehrig's disease or ALS (amyotrophic lateral sclerosis) is caused by the degeneration and death of spinal motor neurons, which carry messages from the spinal cord to the body's muscles. This leads to paralysis of muscles and, eventually, death. Some 30,000 people in the United States suffer from the disease, which has no cu
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