| First Author | Bunting KD | Year | 1998 |
| Journal | Nat Med | Volume | 4 |
| Issue | 1 | Pages | 58-64 |
| PubMed ID | 9427607 | Mgi Jnum | J:45049 |
| Mgi Id | MGI:1101663 | Doi | 10.1038/nm0198-058 |
| Citation | Bunting KD, et al. (1998) Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer [see comments]. Nat Med 4(1):58-64 |
| abstractText | 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 repopulating 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. |
