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Ann N Y Acad Sci. 2011 Dec;1246:131-40. doi: 10.1111/j.1749-6632.2011.06314.x.

Homologous recombination-based gene therapy for the primary immunodeficiencies.

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  • 1Department of Pediatrics, Divisions of Cancer Biology, Hematology/Oncology, Human Gene Therapy, Stanford University, Stanford, California, USA.


The devastating nature of primary immunodeficiencies, the ability to cure primary immunodeficiencies by bone marrow transplantation, the ability of a small number of gene-corrected cells to reconstitute the immune system, and the overall suboptimal results of bone marrow transplantation for most patients with primary immunodeficiencies make the development of gene therapy for this class of diseases important. While there has been clear clinical benefit for a number of patients from viral-based gene therapy strategies, there have also been a significant number of serious adverse events, including the development of leukemia, from the approach. In this review, I discuss the development of nuclease-stimulated, homologous recombination-based approaches as a novel gene therapy strategy for the primary immunodeficiencies.

© 2011 New York Academy of Sciences.

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