Gene therapy for primary immunodeficiencies.
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.
UCL Institute of Child Health, Centre for Immunodeficiency, London WCIN 1EH, United Kingdom.
Christine
C
Rivat
Giorgia
G
Santilli
H Bobby
HB
Gaspar
Adrian J
AJ
Thrasher
D000818:Animals; D002986:Clinical Trials as Topic; D017988:Gammaretrovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods; D005822:Genetic Vectors; D059765:Homologous Recombination; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics / Q000628:therapy*