![]() But the new treatment worked to prevent this from happening by including "insulator" genes that essentially block activation of the adjacent genes to prevent them from turning cancerous ![]() One concern with gene therapy is that, after inserting a gene into people's DNA, genes that are next to the insertion site may turn cancerous, as happened in prior cases where people developed leukemia. Before this infusion, the patients received a low dose of a chemotherapy drug to help make space in their marrow for the new cells to grow. These cells were then infused back into the patients. Then, they used the altered version of HIV to insert a working copy of the IL2RG gene into the bone marrow cells. In the new study, the researchers first collected patients' bone marrow. For example, a gene-therapy treatment in the early 2000s resulted in several patients developing leukemia. Some previous attempts to treat SCID-X1 with gene therapy have had serious side effects. He died at age 12, after receiving his transplant. The name "bubble boy disease" comes from the highly publicized case of David Vetter, who was born in 1971 with SCID-X1, and spent most of his life in a plastic bubble while awaiting a bone-marrow transplant, according to CBS. Bone-marrow transplants from unrelated donors are typically less effective and come with greater risks. But fewer than 20% of patients with SCID-X1 have such a donor available, the authors said. The disease can be essentially cured by a bone-marrow transplant from a sibling that is a match in terms of certain immune system proteins. The condition is rare, likely affecting about 1 in 50,000 to 100,000 newborns. SCID-X1 is caused by a mutation in a gene called IL2RG, which is critical for normal immune function, according to the National Institutes of Health. But they will still need to be monitored for a longer period to determine if the treatment is long-lasting and doesn't cause side effects later in life, the researchers said. ![]() About 16 months after their treatment, the patients are developing normally and have not experienced serious side effects from the therapy.
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