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Blood, Vol. 95 No. 10 (May 15), 2000:
pp. 3071-3077
Stable and functional lymphoid reconstitution of common cytokine
receptor  chain deficient mice by retroviral-mediated gene transfer
Claire Soudais,
Tsujino Shiho,
Lama I. Sharara,
Delphine Guy-Grand,
Tadatsugu Taniguchi,
Alain Fischer, and
James P. Di Santo
From the INSERM U429, Hôpital Necker, Paris, France;
Department of Immunology, University of Tokyo, Tokyo, Japan; and
Unité des Cytokines et Développement Lymphoide, Institut
Pasteur, Paris, France.
Mutations in the gene encoding the common cytokine receptor gamma
chain ( c) are responsible for human X-linked severe
combined immunodeficiency disease (SCIDX1). We have used a
c-deficient mouse model to test the feasibility and
potential toxicity of c gene transfer as a therapy for
SCIDX1. A retrovirus harboring the murine c chain was
introduced into c-deficient bone marrow cells, which
were then transplanted into alymphoid RAG2/c
double-deficient recipient mice. Circulating lymphocytes appeared 4 weeks postgraft and achieved steady-state levels by 8 weeks. The mature
lymphocytes present in the grafted mice had integrated the
c transgene, expressed c transcripts, and
were able to proliferate in response to c-dependent cytokines. The c-transduced animals demonstrated (1)
normal levels of immunoglobulin subclasses, including immunoglobulin G1
(IgG1) and IgG2a (which are severely decreased in
c- mice); (2) the ability to mount an
antigen-specific, T-dependent antibody response showing effective in
vivo T-B cell cooperation, and (3) the presence of gut-associated
cryptopatches and intraepithelial lymphocytes. Importantly, peripheral
B and T cells were still present 47 weeks after a primary graft, and
animals receiving a secondary graft of c-transduced bone
marrow cells demonstrated peripheral lymphoid reconstitution. That
c gene transfer to hematopoietic precursor cells can
correct the immune system abnormalities in c- mice supports the feasibility of in vivo
retroviral gene transfer as a treatment for human SCIDX1.
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