3D genome architecture influences SCID-X1 gene therapy success

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), sometimes called “bubble boy disease,” are born with a defective gene that prevents them from producing immune cells. Gene therapy from St. Jude Children’s Research Hospital restored the immune system in multiple infants with SCID-X1 in 2019 by supplying copies of the corrected gene.