Center for
Biologics Evaluation and Research
Biological
Response Modifiers Advisory Committee
Meeting #33,
October 10, 2002
Bibliography:
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.