Genetic treatment has changed the reality of eight children suffering from a condition they call the "Bubble Boys". These children were born without a strengthened immune system, making any infection a major threat to their lives. Therefore, they live in closed rooms without contact to the outside world. The novelty is that this gene therapy was made by one of the worst enemies of the immune system, the HIV virus, causing AIDS. Researchers at the St. Jude Children's Research Hospital in Memphis engineered this virus so that it did not cause disease and then used it as a vehicle for a gene that patients did not have
– For a long time we did not know what was wrong with that. He just got these infections. (Knowing that he had SCID) It was only heartbreaking – said Kristin Simpson, mother of Omarion. "It's like a normal, healthy baby, I find it incredible."
SCID is caused by a genetic defect that prevents the bone marrow from forming effective versions of the blood cells that make up the immune system, affecting one in every 200,000 newborns Almost all men, without treatment, often kills the baby in the first or second year of life.
The nickname "Bubble Boy" comes from a famous case in the 70s when a Texas boy lived in a hospital for 12 years Germinating a Protective Plastic Bladder Bone marrow transplantation of a genetically compatible sibling can cure SCID, but most people do not have a proper donor.
Gene therapy might be a solution, with some blood cells taken from a patient with the missing gene inserted through modified HIV and the cell be later returned to the body. Before receiving the cells, patients are given a drug to destroy part of the bone marrow so that the modified cells have more room to grow.
When physicians first attempted to use therapy 20 years ago, the treatment had an inadvertent effect on other genes, and some patients subsequently developed leukemia. The new therapy is being supported to reduce this risk.
Source: O Globo