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Correction of interleukin-2 receptor function in X-SCID lymphoblastoid
cells by retrovirally mediated transfer of the gamma-c gene
N Taylor, L Uribe, S Smith, T Jahn, DB Kohn and K Weinberg
Division of Research Immunology and Bone Marrow Transplantation, Childrens
Hospital Los Angeles, California 90027, USA.
X-SCID, the most common form of human SCID, is due to mutations in the
common gamma chain gene (gamma-c) that encodes an essential component of
the cytokine receptors for interleukin-2 (IL-2), IL-4, IL-7, IL-9, and
IL-15. Activation of the Janus family tyrosine kinases Jak1 and Jak3 is
necessary for appropriate signalling through the IL-2 receptor (IL-2R).
Neither Jak1 nor Jak3 was phosphorylated after IL-2 stimulation of an
Epstein-Barr virus-transformed cell line (LCL) from an X-SCID patient with
a gamma-c null mutation. However, we now show that appropriate IL-2R
function can be restored in an X-SCID LCL by transduction of a wild-type
gamma-c gene. A retroviral vector, G1gamma- cSvNa, was constructed and
produced in the PG13 packaging line. Transduced X-SCID LCL expressed the
G1gamma-cSvNa transcript. IL-2 stimulation of the transduced cell line
resulted in appropriate tyrosine phosphorylation of both Jak1 and Jak3.
Thus, retroviral- mediated transduction of normal gamma-c can reconstitute
downstream signalling through the IL-2R in X-SCID cell lines, suggesting
that gene therapy may be a treatment for this disease.
Volume 87,
Issue 8,
pp. 3103-3107,
04/15/1996
Copyright © 1996 by The American Society of Hematology
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