Motor neurone breakthrough
Updated on 31 July 2008
Scientists have succeeded in transforming skin cells from a woman with motor neurone disease into the same kind of nerve cells that are being destroyed by her illness.
The US achievement is being hailed as a major scientific triumph. It could lead to new ways of investigating the causes of the devastating condition, and identifying potential treatments.
It also represents a significant step towards the day when individually tailored stem cell therapies become a reality.
No-one has ever before managed to produce disease-specific stem cells from an individual patient.
The skin cells were taken from an 82-year-old woman with an inherited form of amytrophic lateral sclerosis (ALS), a type of motor neurone disease (MND). ALS occurs when the motor neurones which carry nerve impulses to the body's muscles degenerate, leading to paralysis and ultimately death.
Scientists at the Harvard Stem Cell Institute in Boston and Columbia University in New York used a new technique to produce "pluripotent" stem cells from the patient. These are immature cells with the potential to develop into many different tissues.
Normally they would have been obtained from an early-stage human embryo created through therapeutic cloning. This would involve transferring genetic material from the patient to a human egg whose own nuclear DNA had been removed.
But instead the scientists employed a new technique, first reported by US and Japanese researchers last November, to create induced pluripotent stem (iPS) cells directly from the patient's skin cells.
By inserting four specific genes into the cells, they reprogrammed them to have similar properties as embryonic stem cells. These cells were grown in the laboratory and stimulated to develop into motor neurones that matched the patient.
Professor Chris Henderson, one of the Columbia scientists, said: "Our paper.. shows that we can generate hundreds of millions of motor neurones that are genetically identical to the patient's own neurones. This will be an immense help as we try to uncover the mechanisms behind this disease and screen for drugs that can prolong life."
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