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Updated: Aug 30, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
Published on: May 27, 2011
Gene therapy for inborn and acquired immune deficiency disorders
Barbara C Engel1, Donald B Kohn
1Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital Los Angeles, California, USA.bengel@chla.usc.edu
Abstract:
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.
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