title: Gene therapy for severe combined immunodeficiency: are we there yet?
abstract: Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. In this review, we summarize the advantages and limitations associated with the use of gene therapy to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.
authors.affiliation: INSERM U768 and Université Paris Descartes, Paris, France. m.cavazzana@nck.aphp.fr
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
mesh_terms: D016026:Bone Marrow Transplantation; D002986:Clinical Trials as Topic; D004260:DNA Repair; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D009154:Mutation; D011995:Recombination, Genetic; D012449:Safety; D016511:Severe Combined Immunodeficiency / Q000145:classification / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous