Gene therapy takes boy out of his bubble: GENE therapy has successfully treated a two-year-old boy who has spent most of his life in a sterile "bubble". Rhys Evans has a potentially fatal genetic disorder that left him with no immunity to infection. But now Rhys, born with a defect that causes severe combined immunodeficiency disorder (SCID), is the first British child to be cured with gene therapy for the condition. [...] The gene therapy technique being pioneered at Great Ormond Street is aimed at fixing the genetic fault that prevents a SCID child producing immune system cells. A single faulty gene, called gamma C, is believed to be responsible for the failure of the immune system in boys. The procedure involves removing bone marrow from the patient and treating it with a virus which carries the correct gene into the stem cells. The corrected bone marrow is then infused into the child and allowed to generate immune cells.
That is ... truly incredible. A genetic disease condition that was actually fixed genetically. The body repaired in a way that will be lasting, that means Rhys Evans may get to live a more or less normal life.
It'll be interesting to see what happens if the technique can be replicated for other conditions. I mean, take, for example, genetic deafness or blindness. Fixable shortly after birth or even possibly in the womb. And yet for almost any condition that might be cured this way, there will be protests. From the religious side, people who worry about tinkering with god's work. From another side, constituencies worrying that it shows some sort of prejudice against the handicapped. (Which, in a way, it does ... but isn't it normal for most parents to want the best for their children? To want them to have the best start they can give them? If you can repair certain conditions, why wouldn't you do that? Why wouldn't you make their lives easier?) And there will be all sorts of concerns about the slippery slope, which I can, frankly, share.
But that's for another day. Today, this kid gets to go outdoors and not die from it.Posted by iain at April 04, 2002 01:25 AM