![]() ![]() Genes, made up of DNA, are our body's blueprint.Įach human cell has about 25,000 genes, and each gene's DNA sequence contains instructions on how to build certain enzymes and proteins, each of which has a specific job. The rest must rely on a stem cell transplant from another donor, which is not always possible, and when it is, is less likely to cure SCID-X1 and more likely to lead to severe complications. But only one-fifth of babies have such donors. Until now, the best treatment for SCID-X1 has been a stem cell transplant from a tissue-matched sibling donor. a first for patients with SCID-X1," Dr Mamcarz said. "These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection. Within months of treatment, all 10 children produced functional immune cells for the first time, said Ewelina Mamcarz, a paediatric haematologist-oncologist at St Jude Children's Research Hospital and co-author of the study published in the New England Journal of Medicine on Thursday. In a study of eight children diagnosed with SCID-X1, doctors collected patients' bone marrow and inserted a corrected copy of their mutated gene (the one that caused them to have little immunity) into the DNA of their blood stem cells. Only boys are affected, due to the faulty gene's pattern of inheritance. The disorder, which is estimated to affect about one in 100,000 newborns, is caused by a mutation in the gene that produces a protein essential for normal immune function. A simple infection like a common cold can be fatal. If left untreated, patients with SCID-X1 - the most common type of SCID - rarely live past their first birthday.
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