Media Availability: Gene Therapy Shows Promise for Severe Combined Immunodeficiency

Media Availability: Gene Therapy Shows Promise for Severe Combined Immunodeficiency

Gene Therapy Shows Promise for SCID

An NIAID-funded study suggests that gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening immune disorder. Previous efforts to treat SCID-X1 with gene therapy were initially successful, but approximately one-quarter of the children developed leukemia two to five years after treatment. The new vector appears safer, causing fewer genomic changes that could be linked to leukemia.