Mayo Clinic has signed on as one of three clinical sites for a federally funded project that aims to do something no gene therapy program has managed yet: build a single, reusable system to treat children with rare inherited immune disorders, rather than developing an expensive therapy for each mutation one at a time.
For families raising a child with an inborn error of immunity, the practical stakes are unusually concrete. These are conditions in which a faulty gene leaves a child susceptible to infections that healthy children shrug off. Treatment today often means lifelong antibody infusions, constant infection precautions, or a bone marrow transplant with its own serious risks.
