Update on gene therapy for immunodeficiencies.

Abstract

Primary immune deficiencies (PID) are due to blood cell defects and can be treated with transplantation of normal hematopoietic stem cells (HSC) from another person (allogeneic). Gene therapy in which a patient's autologous HSC are genetically corrected represents an alternative treatment for patients with PID, which could avoid the immunologic risks of allogeneic HSCT and confer similar benefits. Recent clinical trials using gene therapy have led to immune restoration in patients with X-linked severe combined immune deficiency (XSCID), adenosine deaminase (ADA)-deficient SCID and chronic granulomatous disease (CGD). However, severe complications arose in several of the patients in whom the integrated retroviral vectors led to leukoproliferative disorders. New approaches using safer integrating vectors or direct correction of the defective gene underlying the PID are being developed and may lead to safer and effective gene therapy for PID.

• Authors: Donald B Kohn
• Journal: Clinical immunology (Orlando, Fla.) (Clin Immunol) Vol. 135 Issue 2 Pg. 247-54 (May 2010) ISSN: 1521-7035 [Electronic] United States
• PMID: 20071242 (Publication Type: Journal Article, Review)
• Copyright: Copyright 2009 Elsevier Inc. All rights reserved.

Topics

• Clinical Trials as Topic
• Genetic Therapy (methods)
• Genetic Vectors
• Hematopoietic Stem Cell Transplantation (adverse effects)
• Humans
• Immunologic Deficiency Syndromes (genetics, therapy)
• Retroviridae