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Maternal gene therapy trial

Tue, 5th Apr 2016

Kat Arney

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An international group of researchers, including those at UCL and Queen Mary University of London, found that almost all of the 34 pregnant women in a small ethics survey would be happy to participate in a clinical trial for gene therapy if their unborn child is suffering from severe growth problems.

The scientists - all part of a consortium called EVERREST - are currently planning the first ever trial of this kind of maternal gene therapy, but they needed to know whether mothers-to-be will actually be happy to take part. Because if they arenít, then thatís a big problem. The condition to be treated - known as fetal growth restriction - affects up to 8 per cent of all pregnancies, and is usually caused by a lack of blood flow in the womb and placenta.

At the moment, women whose babies are affected by the condition either have to deliver their baby prematurely, to give it the best shot at life, or risk the chance that it may die in the womb. So itís hoped that the new treatment will help to reduce stillbirths and babies dying shortly after birth, as well as cutting long-term complications such as cerebral palsy, diabetes and heart disease in children that do survive.

The gene therapy designed to treat it has been in development since 2013, involving putting a gene that makes a molecule called VEGF directly into the arteries in the motherís womb, where it helps to stabilise the blood vessels and encourage blood to flow. So far itís worked well in animal studies, so the time has come to take it into trials in humans in the next year or so - so fingers crossed for a successful outcome.



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