Rivat, C and Santilli, G and Gaspar, HB and Thrasher, AJ (2012) Gene therapy for primary immunodeficiencies. Hum Gene Ther , 23 (7) 668 - 675. 10.1089/hum.2012.116.
Abstract
For over 40 years, primary immunodeficiencies (PIDs) have featured prominently in the development and refinement of human allogeneic hematopoietic stem cell transplantation. More recently, ex vivo somatic gene therapy using autologous cells has provided remarkable evidence of clinical efficacy in patients without HLA-matched stem cell donors and in whom toxicity of allogeneic procedures is likely to be high. Together with improved preclinical models, a wealth of information has accumulated that has allowed development of safer, more sophisticated technologies and protocols that are applicable to a much broader range of diseases. In this review we summarize the status of these gene therapy trials and discuss the emerging application of similar strategies to other PIDs.
| Type: | Article |
|---|---|
| Title: | Gene therapy for primary immunodeficiencies. |
| Location: | United States |
| Open access status: | An open access publication |
| DOI: | 10.1089/hum.2012.116 |
| Language: | English |
| Additional information: | PMCID: PMC3404418 |
| Keywords: | Animals, Clinical Trials as Topic, Gammaretrovirus, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Homologous Recombination, Humans, Immunologic Deficiency Syndromes |
| UCL classification: | UCL > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Child Health > Department of Infection and Immunity > ICH - Molecular Immunology Unit |
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