Tag: severe combined immunodeficiency

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Gene Therapy Achieves 95% Cure Rate for Bubble Boy Disease

“Bubble boy” disease, or ADA-SCID, is a rare immune disorder caused by ADA gene mutations, leaving kids unable to fight infections without isolation. Affecting 1 in 200,000 births, it disrupts T cells, B cells, and NK cells, often detected via newborn screening. Traditional treatments like enzyme replacement therapy or bone marrow transplants have limitations, including […]