Current progress on gene therapy for primary immunodeficiencies

Gene Ther. 2013 Oct;20(10):963-9. doi: 10.1038/gt.2013.21. Epub 2013 May 30.


Primary immunodeficiencies have played a major role in the development of gene therapy for monogenic diseases of the bone marrow. The last decade has seen convincing evidence of long-term disease correction as a result of ex vivo viral vector-mediated gene transfer into autologous haematopoietic stem cells. The success of these early studies has been balanced by the development of vector-related insertional mutagenic events. More recently the use of alternative vector designs with self-inactivating designs, which have an improved safety profile has led to the initiation of a wave of new studies that are showing early signs of efficacy. The ongoing development of safer vector platforms and gene-correction technologies together with improvements in cell-transduction techniques and optimised conditioning regimes is likely to make gene therapy amenable for a greater number of PIDs. If long-term efficacy and safety are shown, gene therapy will become a standard treatment option for specific forms of PID.

Publication types

  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Genetic Therapy*
  • Genetic Vectors*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immunologic Deficiency Syndromes / therapy*
  • Lentivirus / genetics
  • Severe Combined Immunodeficiency / therapy*
  • Transduction, Genetic
  • Transplantation, Autologous