Media Availability: Gene Therapy Restores Immunity in Children and Young Adults with Rare Immunodeficiency

Media Availability: Gene Therapy Restores Immunity in Children and Young Adults with Rare Immunodeficiency

Gene Therapy for SCID-X1

Findings from an NIAID study show that gene therapy combined with low-dose chemotherapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency, or SCID-X1, a rare genetic immunodeficiency disorder. Initial results, presented at the American Society of Hematology Annual Meeting, show that patients who received the therapy had substantial improvements in immune function and clinical status.