Gene therapy for spinal muscular atrophy could be tested in 2 years

A new treatment for spinal muscular atrophy that kills about 50 British infants every year could begin human trials within two years, after highly encouraging results in animals.  The gene therapy 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.

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