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Science 28 April 2000:
Vol. 288. no. 5466, pp. 669 - 672
DOI: 10.1126/science.288.5466.669
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Reports

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

Marina Cavazzana-Calvo, *123 Salima Hacein-Bey, *123 Geneviève de Saint Basile, 1 Fabian Gross, 2 Eric Yvon, 3 Patrick Nusbaum, 2 Françoise Selz, 1 Christophe Hue, 12 Stéphanie Certain, 1 Jean-Laurent Casanova, 14 Philippe Bousso, 5 Françoise Le Deist, 1 Alain Fischer 124dagger

Severe combined immunodeficiency-X1 (SCID-X1) is an X-linked inherited disorder characterized by an early block in T and natural killer (NK) lymphocyte differentiation. This block is caused by mutations of the gene encoding the gamma c cytokine receptor subunit of interleukin-2, -4, -7, -9, and -15 receptors, which participates in the delivery of growth, survival, and differentiation signals to early lymphoid progenitors. After preclinical studies, a gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gamma c Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells. After a 10-month follow-up period, gamma c transgene-expressing T and NK cells were detected in two patients. T, B, and NK cell counts and function, including antigen-specific responses, were comparable to those of age-matched controls. Thus, gene therapy was able to provide full correction of disease phenotype and, hence, clinical benefit.

1 INSERM Unit 429,
2 Gene Therapy Laboratory,
3 Cell Therapy Laboratory,
4 Unité d'Immunologie et d'Hématologie Pédiatriques, Hôpital Necker, 75743 Paris Cedex 15, France.
5 INSERM Unit 277, Institut Pasteur, 75730 Paris, France.
*   These authors contributed equally to this work.

dagger    To whom correspondence should be addressed at INSERM Unit 429, Hôpital Necker-Enfants Malades, 149 rue de Sèvres, 75743 Paris Cedex 15, France. E-mail: fischer{at}necker.fr

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C. Lutzko, D. Senadheera, D. Skelton, D. Petersen, and D. B. Kohn (2003)
J. Virol. 77, 7341-7351
   Abstract »    Full Text »    PDF »
Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration.
X. Wu, Y. Li, B. Crise, and S. M. Burgess (2003)
Science 300, 1749-1751
   Abstract »    Full Text »    PDF »
Specific transgene expression in human and mouse CD4+ cells using lentiviral vectors with regulatory sequences from the CD4 gene.
G. Marodon, E. Mouly, E. J. Blair, C. Frisen, F. M. Lemoine, and D. Klatzmann (2003)
Blood 101, 3416-3423
   Abstract »    Full Text »    PDF »
Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice.
C. Klein, D. Nguyen, C.-H. Liu, A. Mizoguchi, A. K. Bhan, H. Miki, T. Takenawa, F. S. Rosen, F. W. Alt, R. C. Mulligan, et al. (2003)
Blood 101, 2159-2166
   Abstract »    Full Text »    PDF »
Retroviral vector integration occurs in preferred genomic targets of human bone marrow-repopulating cells.
S. Laufs, B. Gentner, K. Z. Nagy, A. Jauch, A. Benner, S. Naundorf, K. Kuehlcke, B. Schiedlmeier, A. D. Ho, W. J. Zeller, et al. (2003)
Blood 101, 2191-2198
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Retroviral transduction efficiency of G-CSF+SCF-mobilized peripheral blood CD34+ cells is superior to G-CSF or G-CSF+Flt3-L-mobilized cells in nonhuman primates.
P. Hematti, S. E. Sellers, B. A. Agricola, M. E. Metzger, R. E. Donahue, and C. E. Dunbar (2003)
Blood 101, 2199-2205
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T Cell Repertoire Development in Humans with SCID After Nonablative Allogeneic Marrow Transplantation.
M. Sarzotti, D. D. Patel, X. Li, D. A. Ozaki, S. Cao, S. Langdon, R. E. Parrott, K. Coyne, and R. H. Buckley (2003)
J. Immunol. 170, 2711-2718
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High-level ectopic HOXB4 expression confers a profound in vivo competitive growth advantage on human cord blood CD34+ cells, but impairs lymphomyeloid differentiation.
B. Schiedlmeier, H. Klump, E. Will, G. Arman-Kalcek, Z. Li, Z. Wang, A. Rimek, J. Friel, C. Baum, and W. Ostertag (2003)
Blood 101, 1759-1768
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Radiosensitive SCID patients with Artemis gene mutations show a complete B-cell differentiation arrest at the pre-B-cell receptor checkpoint in bone marrow.
J. G. Noordzij, N. S. Verkaik, M. van der Burg, L. R. van Veelen, S. de Bruin-Versteeg, W. Wiegant, J. M. J. J. Vossen, C. M. R. Weemaes, R. de Groot, M. Z. Zdzienicka, et al. (2003)
Blood 101, 1446-1452
   Abstract »    Full Text »    PDF »
Gene therapy of apolipoprotein E-deficient mice using a novel macrophage-specific retroviral vector.
P. J. Gough and E. W. Raines (2003)
Blood 101, 485-491
   Abstract »    Full Text »    PDF »
Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5.
X.-F. Qin, D. S. An, I. S. Y. Chen, and D. Baltimore (2003)
PNAS 100, 183-188
   Abstract »    Full Text »    PDF »
Immunodeficiency Disorders.
M. D. Cooper, L. L. Lanier, M. E. Conley, and J. M. Puck (2003)
Hematology 2003, 314-330
   Abstract »    Full Text »    PDF »


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