Reference Publications
Vector Genotoxicity-Referenced Publications
Baum C, Dullmann J, Li Z, Fehse B, Meyer J, Williams DA, von Kalle C. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15;101(6):2099-2114.
Baum C, von Kalle C, Staal FJ, Li Z, Fehse B, Schmidt M, Weerkamp F, Karlsson S, Wagemaker G, Williams DA. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan;9(1):5-13.
Chandra S, Levran O, Jurickova I, Maas C, Kapur R, Schindler D, Henry R, Milton K, Batish SD, Cancelas JA, Hanenberg H, Auerbach AD, Williams DA. A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov;12(5):976-984.
Kraunus J, Schaumann DH, Meyer J, Modlich U, Fehse B, Brandenburg G, von Laer D, Klump H, Schambach A, Bohne J, Baum C. Self-inactivating retroviral vectors with improved RNA processing. Gene Ther. 2004 Nov;11(21):1568-1578.
Kustikova O, Fehse B, Modlich U, Yang M, Dullmann J, Kamino K, von Neuhoff N, Schlegelberger B, Li Z, Baum C. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science. 2005 May 20;308(5725):1171-1174.
Kustikova OS, Geiger H, Li Z, Brugman MH, Chambers SM, Shaw CA, Pike-Overzet K, de Ridder D, Staal FJ, von Keudell G, Cornils K, Nattamai KJ, Modlich U, Wagemaker G, Goodell MA, Fehse B, Baum C. Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways. Blood. 2007 Mar 1;109(5):1897-1907.
Li Z, Dullmann J, Schiedlmeier B, Schmidt M, von Kalle C, Meyer J, Forster M, Stocking C, Wahlers A, Frank O, Ostertag W, Kuhlcke K, Eckert HG, Fehse B, Baum C. Murine leukemia induced by retroviral gene marking. Science. 2002 Apr 19;296(5567):497.
Modlich U, Bohne J, Schmidt M, von Kalle C, Knoss S, Schambach A, Baum C. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood. 2006 Oct 15;108(8):2545-2553.
Modlich U, Kustikova OS, Schmidt M, Rudolph C, Meyer J, Li Z, Kamino K, von Neuhoff N, Schlegelberger B, Kuehlcke K, Bunting KD, Schmidt S, Deichmann A, von Kalle C, Fehse B, Baum C. Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood. 2005 Jun 1;105(11):4235-4246.
Mohamedali A, Moreau-Gaudry F, Richard E, Xia P, Nolta J, Malik P. Self-inactivating lentiviral vectors resist proviral methylation but do not confer position-independent expression in hematopoietic stem cells. Mol Ther. 2004 Aug;10(2):249-259.
Moreau-Gaudry F, Xia P, Jiang G, Perelman NP, Bauer G, Ellis J, Surinya KH, Mavilio F, Shen CK, Malik P. High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors. Blood. 2001 Nov 1;98(9):2664-2672.
Puthenveetil G, Scholes J, Carbonell D, Qureshi N, Xia P, Zeng L, Li S, Yu Y, Hiti AL, Yee JK, Malik P. Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector. Blood. 2004 Dec 1;104(12):3445-3453.
Schambach A, Mueller D, Galla M, Verstegen MM, Wagemaker G, Loew R, Baum C, Bohne J. Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors. Gene Ther. 2006 Nov;13(21):1524-1533.
Schambach A, Galla M, Modlich U, Will E, Chandra S, Reeves L, Colbert M, Williams DA, von Kalle C, Baum C. Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. Exp Hematol. 2006 May;34(5):588-592.
Schmidt M, Glimm H, Wissler M, Hoffmann G, Olsson K, Sellers S, Carbonaro D, Tisdale JF, Leurs C, Hanenberg H, Dunbar CE, Kiem HP, Karlsson S, Kohn DB, Williams D, Von Kalle C. Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Ann N Y Acad Sci. 2003 May;996(112-121.
Schmidt M, Carbonaro DA, Speckmann C, Wissler M, Bohnsack J, Elder M, Aronow BJ, Nolta JA, Kohn DB, von Kalle C. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med. 2003 Apr;9(4):463-468.
Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Abina SH, Cavazzana-Calvo M, Fischer A. Gene therapy: X-SCID transgene leukaemogenicity. Nature. 2006 Sep 21;443(7109):E5-6; discussion E6-7.
von Kalle C, Baum C, Williams DA. Lenti in red: progress in gene therapy for human hemoglobinopathies. J Clin Invest. 2004 Oct;114(7):889-891.
Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM. Gene therapy: therapeutic gene causing lymphoma. Nature. 2006 Apr 27;440(7088):1123.
Stem and Progenitor Cell Expansion and Selection-Referenced Publications
Cotugno G, Pollock R, Formisano P, Linher K, Beguinot F, Auricchio A. Pharmacological regulation of the insulin receptor signaling pathway mimics insulin action in cells transduced with viral vectors. Hum Gene Ther. 2004 Nov;15(11):1101-1108.
Cornetta K, Croop J, Dropcho E, Abonour R, Kieran MW, Kreissman S, Reeves L, Erickson LC, Williams DA. A pilot study of dose-intensified procarbazine, CCNU, vincristine for poor prognosis brain tumors utilizing fibronectin-assisted, retroviral-mediated modification of CD34+ peripheral blood cells with O6-methylguanine DNA methyltransferase. Cancer Gene Ther. 2006 Sep;13(9):886-895.
Jansen M, Sorg UR, Ragg S, Flasshove M, Seeber S, Williams DA, Moritz T. Hematoprotection and enrichment of transduced cells in vivo after gene transfer of MGMT(P140K) into hematopoietic stem cells. Cancer Gene Ther. 2002 Sep;9(9):737-746.
Meisel R, Bardenheuer W, Strehblow C, Sorg UR, Elmaagacli A, Seeber S, Flasshove M, Moritz T. Efficient protection from methotrexate toxicity and selection of transduced human hematopoietic cells following gene transfer of dihydrofolate reductase mutants. Exp Hematol. 2003 Dec;31(12):1215-1222.
Neff T, Horn PA, Peterson LJ, Thomasson BM, Thompson J, Williams DA, Schmidt M, Georges GE, von Kalle C, Kiem HP. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest. 2003 Nov;112(10):1581-1588.
Rattmann I, Kleff V, Sorg UR, Bardenheuer W, Brueckner A, Hilger RA, Opalka B, Seeber S, Flasshove M, Moritz T. Gene transfer of cytidine deaminase protects myelopoiesis from cytidine analogs in an in vivo murine transplant model. Blood. 2006 Nov 1;108(9):2965-2971.
