Gene therapy for inborn and acquired immune deficiency disorders.
Gene therapy has been under development as a way to correct inborn errors for over 20 years. Immune deficiencies are favorable candidates for gene therapy because of the potential selective advantage of genetically corrected cells in these conditions. Gene therapy for immune deficiencies has been the only application to show incontrovertible benefit in clinical trials to date. Despite the success in treating the underlying disease, there have been two cases of insertional oncogenesis reported in one of these early phase trials. Gene therapy approaches and clinical trials for several inborn as well as acquired immune deficiencies will be reviewed.
Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital Los Angeles, California, USA.bengel@chla.usc.edu
Barbara C
BC
Engel
Donald B
DB
Kohn
D000163:Acquired Immunodeficiency Syndrome / Q000628:therapy*; D015316:Genetic Therapy; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000628:therapy*