New gene therapy offers hope for older kids and adults with 'bubble boy' disease

This study tests a gene therapy for people aged 2 to 50 with X-linked severe combined immunodeficiency (SCID-X1), a condition that leaves them without a working immune system. Participants receive their own blood stem cells that are genetically corrected in a lab, then infused back after mild chemotherapy. The goal is to restore immune function and reduce infections, especially for those who cannot get a perfect bone marrow match.