http://story.news.yahoo.com/news?tmp...ritain_gene_dc
LONDON (Reuters) - British doctors have successfully used gene therapy for the first time to cure a Welsh toddler born without an immune system, the Great Ormond Street Hospital for Children said on Wednesday.
The London hospital said 18-month-old Rhys Evans had been cured of the fatal genetic condition X-linked Severe Combined Immunodeficiency Disease (X-SCID), commonly known as "baby in the bubble" syndrome.
"This is the first time gene therapy has cured a child in Britain," the hospital said in a statement.
The disease meant Rhys was born without an immune system and was highly susceptible to potentially fatal infections.
He was forced to live in a totally sterile environment, isolated from other children, and spent much of his life in hospital.
Doctors removed some of Rhys' bone marrow and genetically modified it to add a correct copy of the faulty gene which caused X-SCID. The marrow was then re-injected into his system.
Dr. Adrian Thrasher, who led the team which carried out the procedure, said the success of Rhys' treatment was very exciting.
"Gene therapy is about turning understanding into real cures for real children," he said.
Rhys' mother Marie said the treatment had made a huge difference for her son.
"We see him now playing with other children and it is just amazing," she told the BBC.
The Evans case, which followed the opening of a gene therapy laboratory at the hospital last September, is one of the few clinically successful examples of gene therapy in the world.
French researchers claimed the first success for gene therapy in 2000 when they treated two infant boys with X-SCID.
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