Gene therapy could prevent illnesses “in the womb”

Serious illnesses such as cystic fibrosis could be prevented in the womb using gene therapy.


In gene therapy, a healthy copy of a faulty gene is put into the body to correct the defect and Dr Simon Waddington, from University College London, said his team had been investigating how such therapies could be delivered in the womb.


Researchers have used mice to show how the treatment could be delivered to babies before they are born and start suffering from health problems. But they warned that while much work was being carried out around the world, such therapies were still at least a decade away from being used in humans.

Dr Waddington said that, via ultrasound techniques, researchers had been able to show how such a treatment, when it is eventually developed, could be sent straight to the lungs of an unborn mouse. Researchers now hope to carry out tests on monkeys.


“With something like cystic fibrosis, there is evidence that damage to the lungs starts in the womb. If we can treat the problem before that starts, that is where it is going to have the most benefit,” Dr Waddington said.

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