title: Occurrence of leukaemia following gene therapy of X-linked SCID.
abstract: Recombinant viral vectors have allowed gene transfer to be developed as a promising approach to the treatment of genetic diseases. Recently, gene therapy of children with X-linked severe combined immune deficiency resulted in impressive levels of immune reconstitution--a triumph that was later overshadowed by the development of leukaemia in two patients. What were the causes of this cancer, and how can the therapeutic benefits of gene therapy be achieved while minimizing risk to the patient?
authors.affiliation: Division of Research Immunology/BMT, Childrens Hospital Los Angeles, USC Keck School of Medicine, 4650 Sunset Boulevard, Los Angeles, California 90027, USA. dkohn@chla.usc.edu
authors.forename: Donald B
authors.initials: DB
authors.lastname: Kohn
authors.forename: Michel
authors.initials: M
authors.lastname: Sadelain
authors.forename: Joseph C
authors.initials: JC
authors.lastname: Glorioso
mesh_terms: D002471:Cell Transformation, Neoplastic; D002986:Clinical Trials as Topic; D040181:Genetic Diseases, X-Linked / Q000150:complications / Q000235:genetics / Q000628:therapy*; D015316:Genetic Therapy / Q000009:adverse effects* / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D007938:Leukemia / Q000209:etiology*; D016511:Severe Combined Immunodeficiency / Q000150:complications / Q000235:genetics / Q000628:therapy*