Scientists have managed to reverse nature’s actions as they rewound skin cells to an embryonic state, creating the first stem cell lines. These stem cells were then used to form neurons, which were genetically similar to the degenerated cells in people with Lou Gehrig's disease.
Technically known as amyotrophic lateral sclerosis, the disease damages the nerve cells in the brain and spinal cord, eventually leading to death.
Now, by going back to the embryonic form of the cells, scientists would be able to observe the development of the disease, which in turn would help them form better treatments.
The cells were taken from an 82-year-old woman and her 89-year-old sister, who share a rare genetic mutation that causes about 2 percent of ALS cases.
In order to avoid embryo destruction and other ethical issues of embryonic stem cell research, researchers used the technique of Japanese stem cell researchers and simply inserted four genes into a patient's cells, reprogramming them into stem cells found in embryonic state.
"Since the cloning of Dolly the sheep and the first derivation of a human embryonic stem cell line by Jamie Thomson some 10 years ago, it's been the hope of scientists . . . to generate stem cell lines that have the genes of a patient," said Kevin Eggan, co-author of the paper and a principal faculty member of the Harvard Stem Cell Institute.
He hopes that this development would possibly help to make these cells in patients suffering from other diseases, like Parkinson's disease, diabetes, or genetic heart problems.
Christopher Henderson, a coauthor of the paper and co-director of the Center for Motor Neuron Biology and Disease at Columbia University, believes that effective cures cannot be developed because of the limited knowledge of the disease.
"We now have in the culture dish cells which have the same genetic makeup as do the ALS patients, and they are the very cells that are affected in the disease. There's no way we could go to an ALS patient and take a sample of their motor neurons," he said.
"Now we have to figure out whether these motor neurons are able to mature," said Dr. Lucie Bruijn, science director and vice president of the ALS Association. "For these to be useful for drug development we need mature motor neurons."
The study was published on Thursday in the online edition of Science.
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