Gene therapy for inherited immunodeficiencies.
Extensive research into gene therapy technologies has produced few clinically relevant results. Advances in the understanding of the genetics of inherited immunologic diseases, gene transfer methodologies, and stem cell manipulation have all contributed to successes in treating X-linked severe combined immunodeficiency and adenosine deaminase deficiency. This review examines the progress and the problems that have arisen, and discusses the improvement and future of gene therapy for primary immunodeficiencies.
Howe
Steven
S
Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London WCIN 1EH, UK. s.howe@ich.ucl.ac.uk
Thrasher
Adrian J
AJ
D000243:Adenosine Deaminase / Q000172:deficiency; D015316:Genetic Therapy / Q000379:methods*; D006412:Hematopoietic Stem Cells; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000473:pathology / Q000628:therapy*; D019891:Leukopoiesis; D016511:Severe Combined Immunodeficiency / Q000473:pathology / Q000628:therapy
D000243:Adenosine Deaminase