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Child with Rare 'Bubble Baby' Syndrome Trials World's First Cure

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Published on Jul 15, 2013

Child with Rare 'Bubble Baby' Syndrome Trials World's First Cure

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A BABY born without an immune system is trialling a world-first gene therapy cure to 're-boot' her body's defence systems and give her the chance of life outside a sterilised environment. Seventeen-month-old Nina Warnell suffers from Severe Combined Immunodeficiency Syndrome (SCID) - an inherited condition known as 'bubble baby' syndrome - which affects just one in 300,000 babies. She has all the appearance of a healthy child but is unable to fight even the mildest germs - meaning even a cough or sneeze could kill her. The family, who were living in Poland when Nina fell ill and was given four months to live, have turned their suburban house in Maidenhead, Berkshire, into a quarantine zone and must screen everyone and everything that enters the house. The radical new treatment - devised by doctors at Great Ormond Street Hospital - has its risks but doting parents Graeme and Aga Warnell have been left with few choices. Nina is missing a gene which produces an enzyme vital to the production of a healthy immune system. A team led by Professor Bobby Gaspar, a consultant in paediatric immunology, are in the process of trialling a new form of gene therapy on the youngster. The doctors have harvested Nina's bone marrow and re-engineered it using a new type of 'reprogrammed virus' to splice the vital gene she is missing into her DNA profile. The re-engineered bone marrow has been re-inserted into her body and they hope a fully functioning immune system will develop. They have been given a 60-90 per cent chance of success but will not know the outcome until Christmas.

Videographer/Director: Richard Grange
Producer: John Balson
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