title: Gene therapy for primary immunodeficiencies.
abstract: Primary immunodeficiencies are a group of disorders that are highly amenable to gene therapy because of their defined pathophysiology and the accessibility of the hematopoietic system to molecular intervention. The development of this new therapeutic modality has been driven by the established morbidity and mortality associated with conventional allogeneic stem cell transplantation, particularly in the human leukocyte antigen-mismatched setting. Recently, several clinical studies have shown that gamma retroviral gene transfer technology can produce major beneficial therapeutic effects, but, as for all cellular and pharmacologic treatment approaches, with a finite potential for toxicity. Newer developments in vector design showing promise in overcoming these issues are likely to establish gene therapy as an efficacious strategy for many forms of primary immunodeficiencies.
authors.affiliation: Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London and Great Ormond Street Hospital for Children NHS Trust, 30 Guilford Street, London WC1N 1EH, UK. a.thrasher@ich.ucl.ac.uk
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency*; D015316:Genetic Therapy / Q000009:adverse effects; 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