*First Gene Therapy Cure 2002-04-14Posted by clype in Science.
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The first success for' gene therapy' in the United Kingdom has restored health to an infant who last year was confined to a sterile room and needed to be kept alive by a ventilator.
Mr.Rhys Evans, now 18 months old, suffered from 'X-chromosome' linked 'Severe Combined Immunodeficiency Disorder', ('S.C.I.D.') popularly known as 'baby in the bubble' syndrome, after the sterile plastic spheres used to protect some babies from infection. He now lives a normal life at home.
At a press conference last week his physicians pronounced themselves 'ecstatic' about the boy's recovery.
'We're very pleased to announce the first successful cure of a patient in the United Kingdom by gene therapy.'
–So said Professor Ms.Christine Kinnon, Director of 'The Centre for Gene Therapy of Childhood Disease' at 'The Institute of Child Health', London, who collaborated with physicians at 'Great Ormond Street Hospital' in treating Rhys. Mr.Adrian Thrasher, consultant paediatric immunologist at 'Great Ormond Street Hospital', was more cautious:
'I'd like to say that Rhys is cured, but we can't say that.
'He will be followed up for years and years.'
Rhys's condition appeared soon after his mother stopped breast feeding him at four months. A series of chest infections developed into 'severe pneumonia'.
'He was skin and bones, too weak to hold his head up,' said his father, Mr.Mark Evans.
The standard treatment for the condition is a 'bone marrow transplant', but Rhys had no obvious donors. An U.S. American donor was finally located at the same time that gene therapy became available.
The parents opted for the new treatment, partly to avoid the 'chemotherapy' that accompanies 'transplantation'.
The treatment began with the extraction and freezing of cells from Rhys's bone marrow. A 'mouse retrovirus' was genetically engineered to carry a healthy copy of the gene that was defective in Rhys.
A bag containing the 'retrovirus' was connected to a 'bag' of his bone marrow. The virus then spliced the healthy gene into the 'stem cells', which were then re-inserted into the bone. Within a few months his 'lymphocyte count' began to climb rapidly.
Three months ago a second, 10 month old baby underwent gene therapy for severe 'combined immunodeficiency disorder' at 'Great Ormond Street Hospital'. That patient shows even more promising results than did Rhys at the same stage, Mr.Thrasher said.