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Retroviral-mediated gene correction for X-linked severe combined
immunodeficiency
F Candotti, JA Johnston, JM Puck, K Sugamura, JJ O'Shea and RM Blaese
Clinical Gene Therapy Branch, NCHGR, National Institutes of Health,
Bethesda, Maryland 20892-1852 USA.
X-linked severe combined immunodeficiency (XSCID) is a lethal disease
caused by a defect in the gene encoding the common gamma chain (gamma- c)
of the receptor for interleukin-2 (IL-2), IL-4, IL-7, IL-9, and IL- 15.
Allogeneic bone marrow transplantation, the current therapy of choice for
this defect, is often complicated by graft-versus-host disease and/or
incomplete reconstitution of B-lymphocyte functions. Correction of the gene
defect at the level of the autologous lymphohematopoietic progenitors could
therefore represent an improvement in the medical management of these
patients. To study the feasibility of a gene therapy approach for XSCID, a
retroviral vector expressing gamma-c was used to transduce Epstein-Barr
virus-transformed B-cell lines derived from patients with XSCID. After
transduction, XSCID cells newly expressed gamma-c on the cell surface at
levels comparable to those observed on B-cell lines obtained from normal
donors. Moreover, the reconstituted gamma-c restored function to the IL- 2
and IL-4 receptors as shown by signal transduction mediated by
phosphorylation of the JAK1 and JAK3 members of the Janus family of
tyrosine kinases and by restoration of cellular proliferation in response
to IL-2.
Volume 87,
Issue 8,
pp. 3097-3102,
04/15/1996
Copyright © 1996 by The American Society of Hematology
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