Gene therapy of primary T cell immunodeficiencies

Gene. 2013 Aug 10;525(2):170-3. doi: 10.1016/j.gene.2013.03.092. Epub 2013 Apr 10.


Gene therapy of severe combined immunodeficiencies has been proven to be effective to provide sustained correction of the T cell immunodeficiencies. This has been achieved for 2 forms of SCID, i.e SCID-X1 (γc deficiency) and adenosine deaminase deficiency. Occurrence of gene toxicity generated by integration of first generation retroviral vectors, as observed in the SCID-X1 trials has led to replace these vectors by self inactivated (SIN) retro(or lenti) viruses that may provide equivalent efficacy with a better safety profile. Results of ongoing clinical studies in SCID as well as in other primary immunodeficiencies, such as the Wiskott Aldrich syndrome, will be thus very informative.

Keywords: ADA; GVHD; Gene therapy; HSC; HSCT; IPS; LV; Lentivirus; PIDs; RV; Retrovirus; SAE; SCID; SIN; Severe combined immune deficiencies; WAS; Wiskott Aldrich syndrome; Wiskott–Aldrich syndrome; adenosine deaminase; graft versus host disease; hematopoietic stem cell; hematopoietic stem cell transplantation; induced pluripotent cells; primary immunodeficiencies; retrovirus; self inactivated; serious adverse event; severe combined immunodeficiency.

Publication types

  • Review

MeSH terms

  • Adenosine Deaminase / deficiency
  • Agammaglobulinemia / therapy
  • Clinical Trials as Topic
  • Genetic Therapy / methods*
  • Genetic Vectors / adverse effects
  • Humans
  • Lentivirus / genetics
  • Retroviridae / genetics
  • Severe Combined Immunodeficiency / genetics
  • Severe Combined Immunodeficiency / therapy*
  • T-Lymphocytes / immunology*
  • Treatment Outcome
  • Wiskott-Aldrich Syndrome / therapy*


  • Adenosine Deaminase

Supplementary concepts

  • Severe combined immunodeficiency due to adenosine deaminase deficiency