Food and Drug Administration

Center for Biologics Evaluation and Research

Cellular, Tissue and Gene Therapies Advisory Committee

Meeting #36

March 4, 2005


 Update Retroviral Vector-Mediated Insertional Mutagenesis




Kelly, P. F.,, 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.,, 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.,, 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,, Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science, 2002. 296: p. 2410-2413.


Gaspar, H.B.,, Gene therapy of x-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. The Lancet, 2004. 364: p. 2181-2187.


Modlich, U.,, Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood First Edition Paper, prepublished online February 15, 2005.