Gene therapy of X-linked severe combined immunodeficiency.

Abstract

This review addresses several questions in the light of the results recently obtained by a gene therapy trial for the treatment of X-linked severe combined immunodeficiency. This primary immunodeficiency, characterized by a complete absence of T and natural killer lymphocytes, appeared as a good model for the application of gene therapy, combining an expected selective advantage for transduced cells, an absence of immunological response to the vector and/or the therapeutic transgene together with accessibility to hematopoietic stem cells. After a brief description of the disease and its physiopathology we summarize the clinical results of the gene therapy trial putting them in perspective with those obtained following allogeneic hematopoietic stem cell transplantation. Definitive conclusions cannot be thrown due to the limited number of gene therapy-treated patients and their relatively short follow-up.

• Authors: Marina Cavazzana-Calvo, Salima Hacein-Bey-Abina, Alain Fischer
• Journal: Current opinion in allergy and clinical immunology (Curr Opin Allergy Clin Immunol) Vol. 2 Issue 6 Pg. 507-9 (Dec 2002) ISSN: 1528-4050 [Print] United States
• PMID: 14752333 (Publication Type: Comparative Study, Journal Article, Review)

Topics

• Bone Marrow Transplantation (methods)
• Female
• Genetic Diseases, X-Linked (diagnosis, therapy)
• Genetic Therapy (methods)
• Humans
• Male
• Prognosis
• Risk Assessment
• Severe Combined Immunodeficiency (genetics, therapy)
• Stem Cell Transplantation (methods)
• Transplantation, Homologous
• Treatment Outcome