Format

Send to

Choose Destination
See comment in PubMed Commons below
Nat Med. 1998 Jan;4(1):58-64.

Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer.

Author information

1
Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

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 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.

Comment in

PMID:
9427607
[Indexed for MEDLINE]
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Loading ...
    Support Center