Severe Combined Immunodeficiency Due to RAG1 Deficiency research hub

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID

This study looks at a new gene therapy for babies and toddlers with a severe immune system problem called RAG1-deficient SCID. It's for those who can't find a matched donor for a standard transplant. The therapy uses the child's own cells, updated with healthy genes.