title: Gene therapy for primary immunodeficiencies: Part 1.
abstract: Over 60 patients affected by SCID due to IL2RG deficiency (SCID-X1) or adenosine deaminase (ADA)-SCID have received hematopoietic stem cell gene therapy in the past 15 years using gammaretroviral vectors, resulting in immune reconstitution and clinical benefit in the majority of them. However, the occurrence of insertional oncogenesis in the SCID-X1 trials has led to the development of new clinical trials based on integrating vectors with improved safety design as well as investigation on new technologies for highly efficient gene targeting and site-specific gene editing. Here we will present the experience and perspectives of gene therapy for SCID-X1 and ADA-SCID and discuss the pros and cons of gene therapy in comparison to allogeneic transplantation.
authors.affiliation: U768 INSERM, 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
authors.forename: Salima
authors.initials: S
authors.lastname: Hacein-Bey-Abina
authors.forename: Alessandro
authors.initials: A
authors.lastname: Aiuti
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics; D000818:Animals; D002986:Clinical Trials as Topic / Q000379:methods / Q000639:trends; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods / Q000639:trends; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D053632:X-Linked Combined Immunodeficiency Diseases / Q000235:genetics / Q000276:immunology / Q000628:therapy
chemical_list: D000243:Adenosine Deaminase