Gene therapy trial offers hope for boys with rare immune disorder

This study tested a gene therapy for boys with X-linked chronic granulomatous disease (X-CGD), a rare condition where white blood cells cannot kill bacteria and fungi, leading to severe infections. The treatment involved taking the patients' own blood stem cells, adding a working copy of the faulty gene, and returning them to the body. The goal was to restore immune function and reduce infections, though the study was terminated early after enrolling only 3 participants.