Gene therapy could replace bone marrow transplants for rare immune disease

This trial tests a gene therapy for FHL3, a rare immune disorder caused by a gene mutation. Instead of a donor stem cell transplant, patients receive their own stem cells that have been corrected with a healthy gene. The goal is to restore immune function while avoiding transplant complications like graft-versus-host disease. Up to 5 patients aged 3 months to 45 years will be enrolled.