title: Gene therapy outpaces haplo for SCID-X1.
abstract: In this issue of Blood, Touzot et al report that autologous gene therapy/hematopoietic stem cell transplantation (HSCT) for infants with X-linked severe combined immune deficiency (SCID-X1) lacking a matched sibling donor may have better outcomes than haploidentical (haplo) HSCT. Because gene therapy represents an autologous transplant, it obviates immune suppression before and after transplant, eliminates risks of graft versus host disease (GVHD), and, as the authors report, led to faster immunological reconstitution after transplant than did haplo transplant.
authors.affiliation: UNIVERSITY OF CALIFORNIA, LOS ANGELES.
authors.forename: Donald B
authors.identifier: http://orcid.org/0000-0003-1840-6087
authors.initials: DB
authors.lastname: Kohn
mesh_terms: D005260:Female; D015316:Genetic Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D053631:Interleukin Receptor Common gamma Subunit / Q000276:immunology*; D008297:Male; D013601:T-Lymphocytes / Q000276:immunology*; D013950:Thymus Gland / Q000276:immunology*; D053632:X-Linked Combined Immunodeficiency Diseases / Q000628:therapy*
chemical_list: D053631:Interleukin Receptor Common gamma Subunit