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Dr. Arthur BurghesMark Henderson / The Times
March 1, 2010 - A new treatment for an incurable wasting disease that kills about 50 British infants every year could begin human trials within two years, after highly encouraging results in animals.
The gene therapy for spinal muscular atrophy (SMA), which aims to correct the faulty DNA that causes the condition, has dramatically prolonged the lives of mice with the mutation, while improving their nerve and muscle function. The treated rodents lived for more than 250 days, which is five times longer than those given any other type of therapy and 16 times longer than untreated animals.
A version of the therapy has also been successfully tested on a monkey, suggesting that the technique is highly likely to work in human beings.
Scientists at Ohio State University, who conducted the study, will apply to start trials on children as soon as they have finished toxicity testing. They hope to treat the first patients within two years.
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