title: Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency.
abstract: The severe combined immunodeficiency caused by the absence of adenosine deaminase (SCID-ADA) was the first monogenic disorder for which gene therapy was developed. Over 30 patients have been treated worldwide using the current protocols, and most of them have experienced clinical benefit; importantly, in the absence of any vector-related complications. In this document, we review the progress made so far in the development and establishment of gene therapy as an alternative form of treatment for ADA-SCID patients.
authors.affiliation: Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, 30, Guilford Street, London WC1N 1EH, UK.
authors.forename: Claudia A
authors.initials: CA
authors.lastname: Montiel-Equihua
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
mesh_terms: D000241:Adenosine / Q000378:metabolism*; D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics* / Q000378:metabolism; D000361:Agammaglobulinemia / Q000235:genetics* / Q000473:pathology / Q000628:therapy*; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000009:adverse effects; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D016254:Mutagenesis, Insertional / Q000379:methods; D009474:Neurons / Q000378:metabolism / Q000473:pathology; D016511:Severe Combined Immunodeficiency / Q000235:genetics* / Q000473:pathology / Q000628:therapy*
chemical_list: D000243:Adenosine Deaminase; D000241:Adenosine