Scientists Turn Skin Cells Into Motor Neurons in ALS Patients

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"We don't at all fully understand [ALS], and it is our lack of understanding of that disease process which we believe is preventing us from developing more effective [treatments]," said Christopher Henderson, a co-author on the paper and co-director of the Center for Motor Neuron Biology and Disease at Columbia University. "Because the disease process is happening in the spinal cord in the central nervous system of patients, we don't at all have access to living examples of the neurons that are undergoing the disease process. . . . No way could we go to ALS patients and take samples of their motor neurons."

The scientists had originally planned to use somatic cell nuclear transfer (SCNT), or "therapeutic cloning," to try to accomplish this feat. That process involves removing the genetic material from a donated human oocyte and replacing it with genetic material from the skin cells of patients. The approach has been hindered by political, ethical and other obstacles.

Instead, researchers decided to take adult skin cells from two elderly sisters (aged 82 and 89) with a genetic form of ALS and reprogrammed them into cells resembling embryonic stem cells using a technique called induced pluripotent stem (iPS) cells. iPS has already been successfully used to reprogram healthy adult cells.

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This study was the first to apply the technique to cells from ill patients.

Those embryonic stem cells were then transformed into motor neurons, although it's not yet clear if the cells will suffer from the same disease process.

Although only about 2 percent of people with ALS suffer from this particular form of the disease, Eggan and Henderson believe the approach has promise for studying other forms of the disease. In fact, the research team is already working on producing similar cell lines from patients with the "sporadic" form of the disease.

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