title: Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID).
abstract: Severe combined immunodeficiencies (SCIDs) appear as optimal disease targets to challenge potential efficacy of gene therapy. Ex vivo, retrovirally mediated gene transfer into hematopoietic progenitor cells has been shown to provide sustained correction of two forms of SCID, that is, SCID-X1 and adenosine deaminase deficiencies. In the former case, however, genotoxicity was observed in a minority of patients as a consequence of retroviral integration into proto-oncogenes loci and transactivation. Design of vectors in which the enhancer element of retroviral LTR has been deleted and an internal promoter added (self-inactivated vectors) could provide both safe and efficient gene transfer as being presently tested.
authors.affiliation: Descartes University of Paris, Paris, France.
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Salima
authors.initials: S
authors.lastname: Hacein-Bey-Abina
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency; D000361:Agammaglobulinemia / Q000628:therapy; D002986:Clinical Trials as Topic; D018014:Gene Transfer Techniques; D005818:Genetic Engineering; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D012190:Retroviridae / Q000235:genetics*; D016511:Severe Combined Immunodeficiency / Q000628:therapy*; D016896:Treatment Outcome; D016662:Virus Integration
chemical_list: D000243:Adenosine Deaminase