Author: ["Kevin D. Bunting","Mark Y. Sangster","James N. Ihle","Brian P. Sorrentino"]
CITE.CC academic search helps you expand the influence of your papers.
Abstract
Janus kinase-3 (JAK3) deficiency has recently been identified as a cause of severe combined immunodeficiency (SCID) in humans. We used a mouse model of Jak3-deficient SCID to test a gene therapy approach for treatment of this disease. Transfer of a Jak3 retroviral vector to repopulat-ing hematopoietic stem cells resulted in increased numbers of T and B lymphocytes, reversal of hypogammaglobulinemia, restoration of T-cell activation upon stimulation with mitogens, and development of an antigen-specific immune response after immunization. Analysis for vector copy number in lymphoid and myeloid populations showed a large in vivo selective advantage for Jak3-expressing lymphoid cells. These results show that gene replacement is a feasible treatment strategy for this disease and that naturally occurring in vivo selection of corrected cells is an important advantage of this approach.Cite this article
Bunting, K., Sangster, M., Ihle, J. et al. Restoration of lymphocyte function in Janus Kinase 3-deficient mice by retroviral-mediated gene transfer. Nat Med 4, 58–64 (1998). https://doi.org/10.1038/nm0198-058 