Food and Drug Administration

Center for Biologics Evaluation and Research

Biological Response Modifiers Advisory Committee

Meeting #33, October 10, 2002






1.          Blaese, R. M., K. W. Culver, A. D. Miller, C. S. Carter, T. Fleisher, M. Clerici, G. Shearer, L. Chang, Y. Chiang, P. Tolstoshev, J. J. Greenblatt, S. A. Rosenberg, H. Klein, M. Berger, C. A. Mullen, W. J. Ramsey, L. Muul, R. A. Morgan, and W. F. Anderson. 1995. T lymphocyte-directed gene therapy for ADA-SCID:  Initial trial results after 4 years. Science 270:475-480.

2.          Bordignon, C., L. D. Notarangelo, N. Nobili, G. Ferrari, G. Casorati, P. Panina, N. Mazzolari, D. Maggioni, C. Rossi, P. Servida, A. G. Ugazio, and F. Mavilio. 1995. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Science 270:470-475.

3.          Buckley, R. H., S. E. Schiff, R. I. Schiff, L. Markert, L. W. Williams, J. L. Roberts, L. A. Myers, and F. E. Ward. 1999. Hematopoietic stem cell transplantation for the treatment of severe combined immunodeficiency. The New England Journal of Medicine 340:508-516.

4.          Cavazzana-Calvo, M., S. Hacein-Bey, G. de Saint Basile, F. Gross, E. Yvon, P. Nusbaum, F. Selz, C. Hue, S. Certain, J. L. Casanova, P. Bousso, F. L. Deist, and A. Fischer. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288:669-72.

5.          Fischer, A. 2000. Severe combined immunodeficiencies (SCID). Clinical Experimental Immunology 122:143-149.

6.          Fischer, A., S. Hacein-Bey, and M. Cavazzano-Calvo. 2002. Gene therapy of severe combined immunodeficiencies. Nature Reviews:  Immunology 2:615-621.

7.          Hacein-Bey-Abina, S., F. Le Deist, F. Carlier, C. Bouneaud, C. Hue, J. De Villartay, A. J. Thrasher, N. Wulffraat, R. Sorensen, S. Dupuis-Girod, A. Fischer, M. Cavazzana-Calvo, E. G. Davies, W. Kuis, W. H. K. Lundlaan, and L. Leiva. 2002. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New England Journal of Medicine 346:1185-1193.

8.       Li, Z., J. Dullmann, B. Schiedlmeier, M. Schmidt, C. v. Kalle, J. Meyer, M. Forster, C. Stocking, A. Wahlers, O. Frank, W. Ostertag, K. Kulcke, H.-G. Eckert, B. Fehse, and C. Baum. 2002. Murine leukemia induced by retroviral gene marking. Science 296:497.

9.          Myers, L. A., D. D. Patel, J. M. Puck, and R. H. Buckley. 2002. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 99:872-878.

10.          Rabbitts, T. H. 1998. LMO T-cell translocation oncogenes typify genes activated by chromosomal translocations that alter transcription and developmental processes. Genes and Development 12:2651-2657.