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Clin Immunol. 2010 May;135(2):247-54. doi: 10.1016/j.clim.2009.12.003. Epub 2010 Jan 13.

Update on gene therapy for immunodeficiencies.

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Departments of Microbiology, Immunology and Immunology and Pediatrics, University of California, Los Angeles, 290D Biomedical Sciences Research Building, 615 Charles E. Young Drive South, Los Angeles, CA 90095, USA.


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.

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