title: Gene therapy of primary T cell immunodeficiencies.
abstract: Gene therapy of severe combined immunodeficiencies has been proven to be effective to provide sustained correction of the T cell immunodeficiencies. This has been achieved for 2 forms of SCID, i.e SCID-X1 (γc deficiency) and adenosine deaminase deficiency. Occurrence of gene toxicity generated by integration of first generation retroviral vectors, as observed in the SCID-X1 trials has led to replace these vectors by self inactivated (SIN) retro(or lenti) viruses that may provide equivalent efficacy with a better safety profile. Results of ongoing clinical studies in SCID as well as in other primary immunodeficiencies, such as the Wiskott Aldrich syndrome, will be thus very informative.
authors.affiliation: INSERM U768, Paris, France. alain.fischer@inserm.fr
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; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000009:adverse effects; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D012190:Retroviridae / Q000235:genetics; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D013601:T-Lymphocytes / Q000276:immunology*; D016896:Treatment Outcome; D014923:Wiskott-Aldrich Syndrome / Q000628:therapy*
chemical_list: D000243:Adenosine Deaminase
keywords: ADA; GVHD; Gene therapy; HSC; HSCT; IPS; LV; Lentivirus; PIDs; RV; Retrovirus; SAE; SCID; SIN; Severe combined immune deficiencies; WAS; Wiskott Aldrich syndrome; Wiskott–Aldrich syndrome; adenosine deaminase; graft versus host disease; hematopoietic stem cell; hematopoietic stem cell transplantation; induced pluripotent cells; primary immunodeficiencies; retrovirus; self inactivated; serious adverse event; severe combined immunodeficiency