Gene fix trial aims to tame rare immune disease

This study tests a gene therapy for people with X-linked chronic granulomatous disease (X-CGD), a genetic condition that weakens the immune system and leads to severe infections. Researchers take the patient's own stem cells, add a working copy of the faulty gene, and return the cells to the patient after mild chemotherapy. The goal is to restore immune function and reduce infection risk. Up to 10 participants aged 3 to 60 will be followed for 2 years, then long-term for 13 more years.