title: Gene therapy for severe combined immune deficiency.
abstract: Infants born with severe combined immune deficiencies are prone to life-threatening infections and, without treatment, do not survive beyond the first year of life. Haematopoietic stem cell transplantation from a fully matched donor offers the possibility of cure. In the absence of a suitable matched donor, haploidentical transplants from a parental donor may be undertaken, but these are associated with more complications and lower success rates. Recently, an alternative therapeutic option based on retroviral gene delivery has been used to correct X-linked severe combined immune deficiency (SCID-X1) and adenosine deaminase deficiency. Clinical trials have established that in situations where ex vivo gene transfer into haematopoietic progenitor cells confers a strong selective advantage, the procedure is a feasible alternative to haploidentical transplantation, with favourable kinetics of immune reconstitution.
authors.affiliation: Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK. w.qasim@ich.ucl.ac.uk
authors.forename: Waseem
authors.initials: W
authors.lastname: Qasim
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
mesh_terms: D000243:Adenosine Deaminase / Q000235:genetics; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D007223:Infant; D008954:Models, Biological; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D053632:X-Linked Combined Immunodeficiency Diseases / Q000235:genetics / Q000628:therapy
chemical_list: D000243:Adenosine Deaminase