title: Evolving Gene Therapy in Primary Immunodeficiency.
abstract: Prior to the first successful bone marrow transplant in 1968, patients born with severe combined immunodeficiency (SCID) invariably died. Today, with a widening availability of newborn screening, major improvements in the application of allogeneic procedures, and the emergence of successful hematopoietic stem and progenitor cell (HSC/P) gene therapy, the majority of these children can be identified and cured. Here, we trace key steps in the development of clinical gene therapy for SCID and other primary immunodeficiencies (PIDs), and review the prospects for adoption of new targets and technologies.
authors.affiliation: Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 3JH, UK; University College London Great Ormond Street Institute of Child Health, London WC1N 1EH, UK. Electronic address: a.thrasher@ucl.ac.uk.
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
authors.affiliation: Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School and Harvard Stem Cell Institute, 300 Longwood Avenue, Boston, MA 02115, USA. Electronic address: dawilliams@childrens.harvard.edu.
authors.forename: David A
authors.initials: DA
authors.lastname: Williams
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics* / Q000276:immunology; D002986:Clinical Trials as Topic; D017988:Gammaretrovirus / Q000235:genetics / Q000276:immunology; D015870:Gene Expression; D015316:Genetic Therapy / Q000266:history / Q000379:methods*; D005822:Genetic Vectors / Q000737:chemistry / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D049673:History, 20th Century; D049674:History, 21st Century; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D016086:Lentivirus / Q000235:genetics / Q000276:immunology; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D014184:Transplantation, Homologous
chemical_list: D000243:Adenosine Deaminase
keywords: gene therapy; immunodeficiencies; retrovirus