Food and Drug Administration
Center for Biologics Evaluation and Research
Cellular, Tissue and Gene Therapies Advisory Committee
March 4, 2005
Update Retroviral Vector-Mediated Insertional Mutagenesis
Kelly, P. F., et.al., Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34 positive cells marked with a RD114 pseudotyped oncoretroviral vector. Blood Cells, Molecules and Diseases, 2003. 30: p. 132-143.
Hacein-Bey-Abina, S., et.al., LM02-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science, 2003. 302: p. 415-419.
Hacein-Bey-Abina, S., et.al., Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med, 2002. 346(16): p. 1185-1193.
Dave, U.P., Jenkins, N.A., Copeland, N.G., Gene therapy insertional mutagenesis insights. Science, 2004. 303: p. 333.
Aiuti, A, et.al., Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science, 2002. 296: p. 2410-2413.
Gaspar, H.B., et.al., Gene therapy of x-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. The Lancet, 2004. 364: p. 2181-2187.
Modlich, U., et.al., Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood First Edition Paper, prepublished online February 15, 2005.