title: Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.
authors.affiliation: Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Karin
authors.initials: K
authors.lastname: Pike-Overzet
authors.affiliation: Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
authors.forename: Christopher
authors.initials: C
authors.lastname: Baum
authors.affiliation: Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Robbert G M
authors.initials: RG
authors.lastname: Bredius
authors.affiliation: Département de Biothérapie, Hôpital Necker-Enfants Malades, Paris, France.
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana
authors.affiliation: Department of Pediatrics, Erasmus Medical Center, Rotterdam, The Netherlands.
authors.forename: Gert-Jan
authors.initials: GJ
authors.lastname: Driessen
authors.affiliation: Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Willem E
authors.initials: WE
authors.lastname: Fibbe
authors.affiliation: Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom.
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
authors.affiliation: Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Rob C
authors.initials: RC
authors.lastname: Hoeben
authors.affiliation: Département de Biothérapie, Hôpital Necker-Enfants Malades, Paris, France.
authors.forename: Chantal
authors.initials: C
authors.lastname: Lagresle-Peyrou
authors.affiliation: Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Arjan
authors.initials: A
authors.lastname: Lankester
authors.affiliation: Department of Pharmacy, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Pauline
authors.initials: P
authors.lastname: Meij
authors.affiliation: Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
authors.forename: Axel
authors.initials: A
authors.lastname: Schambach
authors.affiliation: Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom.
authors.forename: Adrian
authors.initials: A
authors.lastname: Thrasher
authors.affiliation: Department of Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.
authors.forename: Jacques J M
authors.initials: JJ
authors.lastname: Van Dongen
authors.affiliation: Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Jaap-Jan
authors.initials: JJ
authors.lastname: Zwaginga
authors.affiliation: Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands. Electronic address: f.j.t.staal@lumc.nl.
authors.forename: Frank J T
authors.initials: FJ
authors.lastname: Staal
mesh_terms: D000818:Animals; D005260:Female; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics*; D018398:Homeodomain Proteins / Q000235:genetics*; D006801:Humans; D016086:Lentivirus / Q000235:genetics*; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000235:genetics* / Q000628:therapy*
chemical_list: D018398:Homeodomain Proteins