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Breakthrough Lou Gehrig's disease research

01/08/2008

Scientists have for the first time managed to transform skin cells into motor neurons - cells in the nervous system that control muscle movement.

They hope this process could one day be used to create tailor-made cells to treat amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease.

This is a progressive disease that harms motor neurons, leading to their ultimate death.

When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralysed.

Scientists at Harvard University and Columbia University took skin cells from a chronically-ill patient with ALS and reprogrammed then into a stem cell-like state (cells that can turn into other cells in the body).

They then coaxed them into specific cell types that would be needed to understand and treat ALS.

The researchers, publishing their findings in the journal Science, say that although there are hurdles to overcome before cell replacement therapies are available, the new cells can be used to study a patient's motor neurons in the laboratory.

"Up until now, it's been impossible to get access to the neurons affected by ALS and, although everyone was excited by the potential of the new technology, it was uncertain that we would be able to obtain them from patients' skin cells," said co-author Dr Chris Henderson.

"Our paper now shows that we can generate hundreds of millions of motor neurons that are genetically identical to a patient's own neurons. This will be an immense help as we try to uncover the mechanisms behind this disease and screen for drugs that can prolong life."