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Vectorology / Biosafety

Further publications:   Gene Therapy     Regenerative Medicine     Hematopoiesis


Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes.

Labenski V, Suerth JD, Barczak E, Heckl D, Levy C, Bernadin O, Charpentier E, Williams DA, Fehse B, Verhoeyen E, Schambach A.

Biomaterials. 2016 Aug;97:97-109. doi: 10.1016/j.biomaterials.2016.04.019.


Development of Inducible Molecular Switches Based on All-in-One Lentiviral Vectors Equipped with Drug Controlled FLP Recombinase.

Maetzig T, Schambach A.

Methods Mol Biol. 2016;1448:23-39. doi: 10.1007/978-1-4939-3753-0_2.


Transduction of Murine Hematopoietic Stem Cells with Tetracycline-regulated Lentiviral Vectors.

Stahlhut M, Schambach A, Kustikova OS.

Methods Mol Biol. 2016;1448:65-76. doi: 10.1007/978-1-4939-3753-0_5.


Comparison of tetracycline-regulated promoters in lentiviral-based vectors in murine transplantation studies.

Stahlhut M, Ha TC, Morgan M, Schambach A, Kustikova OS.

Curr Gene Ther. 2016 Oct 13. [Epub ahead of print]


Retroviral Vectors for Cancer Gene Therapy.

Schambach A, Morgan M.

Recent Results Cancer Res. 2016;209:17-35. doi: 10.1007/978-3-319-42934-2_2.


Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform.

Hübner J, Hoseini SS, Suerth JD, Hoffmann D, Maluski M, Herbst J, Maul H, Ghosh A, Eiz-Vesper B, Yuan Q, Ott M, Heuser M, Schambach A, Sauer MG.

Mol Ther. 2016 Aug;24(7):1216-26. doi: 10.1038/mt.2016.89.


In Vivo Killing Capacity of Cytotoxic T Cells Is Limited and Involves Dynamic Interactions and T Cell Cooperativity.

Halle S, Keyser KA, Stahl FR, Busche A, Marquardt A, Zheng X, Galla M, Heissmeyer V, Heller K, Boelter J, Wagner K, Bischoff Y, Martens R, Braun A, Werth K, Uvarovskii A, Kempf H, Meyer-Hermann M, Arens R, Kremer M, Sutter G, Messerle M, Förster R.

Immunity. 2016 Feb 16;44(2):233-45. doi: 10.1016/j.immuni.2016.01.010.


Viral and Synthetic RNA Vector Technologies and Applications.

Schott JW, Morgan M, Galla M, Schambach A.

Mol Ther. 2016 Sep;24(9):1513-27. doi: 10.1038/mt.2016.143. Review.


Transcriptionally regulated and nontoxic delivery of the hyperactive Sleeping Beauty Transposase.

Cocchiarella F, Latella MC, Basile V, Miselli F, Galla M, Imbriano C, Recchia A.

Mol Ther Methods Clin Dev. 2016 Jun 15;3:16038. doi: 10.1038/mtm.2016.38.


Engineering the AAV capsid to optimize vector-host-interactions.

Büning H, Huber A, Zhang L, Meumann N, Hacker U.

Curr Opin Pharmacol. 2015 Oct;24:94-104. doi: 10.1016/j.coph.2015.08.002. Review.


Surface-Engineered Viral Vectors for Selective and Cell Type-Specific Gene Delivery.

Buchholz CJ, Friedel T, Büning H.

Trends Biotechnol. 2015 Dec;33(12):777-90. doi: 10.1016/j.tibtech.2015.09.008. Review.


Adeno-Associated Virus Type 2 Rep68 Can Bind to Consensus Rep-Binding Sites on the Herpes Simplex Virus 1 Genome.

Seyffert M, Glauser DL, Tobler K, Georgiev O, Vogel R, Vogt B, Agúndez L, Linden M, Büning H, Ackermann M, Fraefel C.

J Virol. 2015 Nov;89(21):11150-8. doi: 10.1128/JVI.01370-15.


Impact of the MRN Complex on Adeno-Associated Virus Integration and Replication during Coinfection with Herpes Simplex Virus 1.

Millet R, Jolinon N, Nguyen XN, Berger G, Cimarelli A, Greco A, Bertrand P, Odenthal M, Büning H, Salvetti A.

J Virol. 2015 Jul;89(13):6824-34. doi: 10.1128/JVI.00171-15.


Efficient generation of gene-modified human natural killer cells via alpharetroviral vectors.

Suerth JD, Morgan MA, Kloess S, Heckl D, Neudörfl C, Falk CS, Koehl U, Schambach A.

J Mol Med (Berl). 2015 Aug 25. [Epub ahead of print]


Lentiviral vector system for coordinated constitutive and drug controlled tetracycline-regulated gene co-expression.

Stahlhut M, Schwarzer A, Eder M, Yang M, Li Z, Morgan M, Schambach A, Kustikova OS.

Biomaterials. 2015 Sep;63:189-201. doi: 10.1016/j.biomaterials.2015.06.022. Epub 2015 Jun 16.


Deoxycytidine-kinase knockdown as a novel myeloprotective strategy in the context of fludarabine, cytarabine or cladribine therapy.

Lachmann N, Czarnecki K, Brennig S, Phaltane R, Heise M, Heinz N, Kempf H, Dilloo D, Kaever V, Schambach A, Heuser M, Moritz T.

Leukemia. 2015 Apr 29. doi: 10.1038/leu.2015.108. [Epub ahead of print] No abstract available.


Deciphering the impact of parameters influencing transgene expression kinetics after repeated cell transduction with integration-deficient retroviral vectors.

Schott JW, Jaeschke NM, Hoffmann D, Maetzig T, Ballmaier M, Godinho T, Cathomen T, Schambach A.

Cytometry A. 2015 May;87(5):405-18. doi: 10.1002/cyto.a.22650. Epub 2015 Feb 27.


Inducible T-cell receptor expression in precursor T cells for leukemia control.

Hoseini SS, Hapke M, Herbst J, Wedekind D, Baumann R, Heinz N, Schiedlmeier B, Vignali DA, van den Brink MR, Schambach A, Blazar BR, Sauer MG.

Leukemia. 2015 Jul;29(7):1530-42. doi: 10.1038/leu.2015.20. Epub 2015 Feb 5.


A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells.

Müller-Kuller U, Ackermann M, Kolodziej S, Brendel C, Fritsch J, Lachmann N, Kunkel H, Lausen J, Schambach A, Moritz T, Grez M.

Nucleic Acids Res. 2015 Feb 18;43(3):1577-92. doi: 10.1093/nar/gkv019. Epub 2015 Jan 20.


Alpharetroviral vectors: from a cancer-causing agent to a useful tool for human gene therapy.

Suerth JD, Labenski V, Schambach A.

Viruses. 2014 Dec 5;6(12):4811-38. doi: 10.3390/v6124811. Review.


Toward a safer integration profile of MLV-based retroviral vectors.

Schambach A.

Mol Ther. 2014 Aug;22(8):1405-6. doi: 10.1038/mt.2014.124. No abstract available.


Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.

Moiani A, Suerth JD, Gandolfi F, Rizzi E, Severgnini M, De Bellis G, Schambach A, Mavilio F.

Genes (Basel). 2014 May 16;5(2):415-29. doi: 10.3390/genes5020415.


All-in-One inducible lentiviral vector systems based on drug controlled FLP recombinase.

Maetzig T, Kuehle J, Schwarzer A, Turan S, Rothe M, Chaturvedi A, Morgan M, Ha TC, Heuser M, Hammerschmidt W, Baum C, Schambach A.

Biomaterials. 2014 May;35(14):4345-56. doi: 10.1016/j.biomaterials.2014.01.057. Epub 2014 Feb 14.


Modified lentiviral LTRs allow Flp recombinase-mediated cassette exchange and in vivo tracing of "factor-free" induced pluripotent stem cells.

Kuehle J, Turan S, Cantz T, Hoffmann D, Suerth JD, Maetzig T, Zychlinski D, Klein C, Steinemann D, Baum C, Bode J, Schambach A.

Mol Ther. 2014 May;22(5):919-28. doi: 10.1038/mt.2014.4. Epub 2014 Jan 17.


Biosafety challenges for use of lentiviral vectors in gene therapy.

Rothe M, Modlich U, Schambach A.

Curr Gene Ther. 2013 Dec;13(6):453-68. Review.


Biosafety features of lentiviral vectors.

Schambach A, Zychlinski D, Ehrnstroem B, Baum C.

Hum Gene Ther. 2013 Feb;24(2):132-42. doi: 10.1089/hum.2012.229. Review.


Retrovirus-based mRNA transfer for transient cell manipulation.

Galla M, Schambach A, Baum C.

Methods Mol Biol. 2013;969:139-61. doi: 10.1007/978-1-62703-260-5_10.


Efficient in vivo regulation of cytidine deaminase expression in the haematopoietic system using a doxycycline-inducible lentiviral vector system.

Lachmann N, Brennig S, Pfaff N, Schermeier H, Dahlmann J, Phaltane R, Gruh I, Modlich U, Schambach A, Baum C, Moritz T.

Gene Ther. 2013 Mar;20(3):298-307. doi: 10.1038/gt.2012.40. Epub 2012 May 17.


Vectorizing mRNA and proteins.

Schambach A, Baum C.

Curr Gene Ther. 2012 Oct;12(5):345-6. No abstract available.


Genetic modification of lymphocytes by retrovirus-based vectors.

Suerth JD, Schambach A, Baum C.

Curr Opin Immunol. 2012 Oct;24(5):598-608. doi: 10.1016/j.coi.2012.08.007. Epub 2012 Sep 17. Review.


Retroviral protein transfer: falling apart to make an impact.

Maetzig T, Baum C, Schambach A.

Curr Gene Ther. 2012 Oct;12(5):389-409. Review.


Alpharetroviral self-inactivating vectors: long-term transgene expression in murine hematopoietic cells and low genotoxicity.

Suerth JD, Maetzig T, Brugman MH, Heinz N, Appelt JU, Kaufmann KB, Schmidt M, Grez M, Modlich U, Baum C, Schambach A.

Mol Ther. 2012 May;20(5):1022-32. doi: 10.1038/mt.2011.309. Epub 2012 Feb 14.


Pseudotype-independent nonspecific uptake of gammaretroviral and lentiviral particles in human cells.

Voelkel C, Galla M, Dannhauser PN, Maetzig T, Sodeik B, Schambach A, Baum C.

Hum Gene Ther. 2012 Mar;23(3):274-86. doi: 10.1089/hum.2011.011. Epub 2012 Jan 12.


Avoiding cytotoxicity of transposases by dose-controlled mRNA delivery.

Galla M, Schambach A, Falk CS, Maetzig T, Kuehle J, Lange K, Zychlinski D, Heinz N, Brugman MH, Göhring G, Izsvák Z, Ivics Z, Baum C.

Nucleic Acids Res. 2011 Sep 1;39(16):7147-60. doi: 10.1093/nar/gkr384. Epub 2011 May 23. Erratum in: Nucleic Acids Res. 2012 Jan;40(2):939.


Gammaretroviral vectors: biology, technology and application.

Maetzig T, Galla M, Baum C, Schambach A.

Viruses. 2011 Jun;3(6):677-713. doi: 10.3390/v3060677. Epub 2011 Jun 3. Review.


Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy.

Avedillo Díez I, Zychlinski D, Coci EG, Galla M, Modlich U, Dewey RA, Schwarzer A, Maetzig T, Mpofu N, Jaeckel E, Boztug K, Baum C, Klein C, Schambach A.

Mol Pharm. 2011 Oct 3;8(5):1525-37. doi: 10.1021/mp200132u. Epub 2011 Aug 31.


Viral and non-viral approaches for transient delivery of mRNA and proteins.

Schott JW, Galla M, Godinho T, Baum C, Schambach A.

Curr Gene Ther. 2011 Oct;11(5):382-98. Review.


Retroviral and transposon-based tet-regulated all-in-one vectors with reduced background expression and improved dynamic range.

Heinz N, Schambach A, Galla M, Maetzig T, Baum C, Loew R, Schiedlmeier B.

Hum Gene Ther. 2011 Feb;22(2):166-76. doi: 10.1089/hum.2010.099. Epub 2010 Dec 19.


Multiplexing RMCE: versatile extensions of the Flp-recombinase-mediated cassette-exchange technology.

Turan S, Kuehle J, Schambach A, Baum C, Bode J.

J Mol Biol. 2010 Sep 10;402(1):52-69. doi: 10.1016/j.jmb.2010.07.015. Epub 2010 Jul 19.


Self-inactivating alpharetroviral vectors with a split-packaging design.

Suerth JD, Maetzig T, Galla M, Baum C, Schambach A.

J Virol. 2010 Jul;84(13):6626-35. doi: 10.1128/JVI.00182-10. Epub 2010 Apr 21.


Protein transduction from retroviral Gag precursors.

Voelkel C, Galla M, Maetzig T, Warlich E, Kuehle J, Zychlinski D, Bode J, Cantz T, Schambach A, Baum C.

Proc Natl Acad Sci U S A. 2010 Apr 27;107(17):7805-10. doi: 10.1073/pnas.0914517107. Epub 2010 Apr 12.


Minicircle performance depending on S/MAR-nuclear matrix interactions.

Broll S, Oumard A, Hahn K, Schambach A, Bode J.

J Mol Biol. 2010 Feb 5;395(5):950-65. doi: 10.1016/j.jmb.2009.11.066. Epub 2009 Dec 29.


Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors.

Maetzig T, Galla M, Brugman MH, Loew R, Baum C, Schambach A.

Gene Ther. 2010 Mar;17(3):400-11. doi: 10.1038/gt.2009.129. Epub 2009 Oct 22.


Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis.

Kustikova OS, Schiedlmeier B, Brugman MH, Stahlhut M, Bartels S, Li Z, Baum C.

Mol Ther. 2009 Sep;17(9):1537-47. doi: 10.1038/mt.2009.134. Epub 2009 Jun 16.


Retroviral insertion site analysis in dominant haematopoietic clones.

Kustikova OS, Modlich U, Fehse B.

Methods Mol Biol. 2009;506:373-90. doi: 10.1007/978-1-59745-409-4_25.


Design and production of retro- and lentiviral vectors for gene expression in hematopoietic cells.

Schambach A, Swaney WP, van der Loo JC.

Methods Mol Biol. 2009;506:191-205. doi: 10.1007/978-1-59745-409-4_14.


Retroviral integration site analysis in hematopoietic stem cells.

Kustikova OS, Baum C, Fehse B.

Methods Mol Biol. 2008;430:255-67. doi: 10.1007/978-1-59745-182-6_18.


Clinical application of lentiviral vectors - concepts and practice.

Schambach A, Baum C.

Curr Gene Ther. 2008 Dec;8(6):474-82. Review.


Physiological promoters reduce the genotoxic risk of integrating gene vectors.

Zychlinski D, Schambach A, Modlich U, Maetzig T, Meyer J, Grassman E, Mishra A, Baum C.

Mol Ther. 2008 Apr;16(4):718-25. doi: 10.1038/mt.2008.5. Epub 2008 Mar 4.


Cellular restriction of retrovirus particle-mediated mRNA transfer.

Galla M, Schambach A, Towers GJ, Baum C.

J Virol. 2008 Mar;82(6):3069-77. doi: 10.1128/JVI.01880-07. Epub 2008 Jan 16.


Vector design for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells.

Schambach A, Baum C.

DNA Repair (Amst). 2007 Aug 1;6(8):1187-96. Epub 2007 May 7. Review.


Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors.

Schambach A, Galla M, Maetzig T, Loew R, Baum C.

Mol Ther. 2007 Jun;15(6):1167-73. Epub 2007 Apr 3.


Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways.

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.

Blood. 2007 Mar 1;109(5):1897-907. Epub 2006 Nov 21.


Insertional mutagenesis by replication-deficient retroviral vectors encoding the large T oncogene.

Li Z, Kustikova OS, Kamino K, Neumann T, Rhein M, Grassman E, Fehse B, Baum C.

Ann N Y Acad Sci. 2007 Jun;1106:95-113. Epub 2007 Mar 29.


New way of regulating alternative splicing in retroviruses: the promoter makes a difference.

Bohne J, Schambach A, Zychlinski D.

J Virol. 2007 Apr;81(7):3652-6. Epub 2007 Jan 17.


Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors.

Schambach A, Mueller D, Galla M, Verstegen MM, Wagemaker G, Loew R, Baum C, Bohne J.

Gene Ther. 2006 Nov;13(21):1524-33. Epub 2006 Jun 8.


Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research.

Schambach A, Galla M, Modlich U, Will E, Chandra S, Reeves L, Colbert M, Williams DA, von Kalle C, Baum C.

Exp Hematol. 2006 May;34(5):588-92.


Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors.

Baum C, Kustikova O, Modlich U, Li Z, Fehse B.

Hum Gene Ther. 2006 Mar;17(3):253-63. Review.


Retrovirus vectors: toward the plentivirus?

Baum C, Schambach A, Bohne J, Galla M.

Mol Ther. 2006 Jun;13(6):1050-63. Epub 2006 Apr 24. Review.


Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression.

Schambach A, Bohne J, Baum C, Hermann FG, Egerer L, von Laer D, Giroglou T.

Gene Ther. 2006 Apr;13(7):641-5.


Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells.

Schambach A, Bohne J, Chandra S, Will E, Margison GP, Williams DA, Baum C.

Mol Ther. 2006 Feb;13(2):391-400. Epub 2005 Oct 12.

2000 - 2005

Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking.

Kustikova O, Fehse B, Modlich U, Yang M, Düllmann J, Kamino K, von Neuhoff N, Schlegelberger B, Li Z, Baum C.

Science. 2005 May 20;308(5725):1171-4.


Self-inactivating retroviral vectors with improved RNA processing.

Kraunus J, Schaumann DH, Meyer J, Modlich U, Fehse B, Brandenburg G, von Laer D, Klump H, Schambach A, Bohne J, Baum C.

Gene Ther. 2004 Nov;11(21):1568-78.


Predictable and efficient retroviral gene transfer into murine bone marrow repopulating cells using a defined vector dose.

Li Z, Schwieger M, Lange C, Kraunus J, Sun H, van den Akker E, Modlich U, Serinsöz E, Will E, von Laer D, Stocking C, Fehse B, Schiedlmeier B, Baum C.

Exp Hematol. 2003 Dec;31(12):1206-14.


Side effects of retroviral gene transfer into hematopoietic stem cells.

Baum C, Düllmann J, Li Z, Fehse B, Meyer J, Williams DA, von Kalle C.

Blood. 2003 Mar 15;101(6):2099-114. Epub 2003 Jan 2. Review.


Upstream conserved sequences of mouse leukemia viruses are important for high transgene expression in lymphoid and hematopoietic cells.

Wahlers A, Kustikova O, Zipfel PF, Itoh K, Koester M, Heberlein C, Li Z, Schiedlmeier B, Skerka C, Fehse B, Baum C.

Mol Ther. 2002 Sep;6(3):313-20. Erratum in: Mol Ther. 2002 Oct;6(4):563..


Retroviral vector-mediated expression of HoxB4 in hematopoietic cells using a novel coexpression strategy.

Klump H, Schiedlmeier B, Vogt B, Ryan M, Ostertag W, Baum C.

Gene Ther. 2001 May;8(10):811-7.


Improved post-transcriptional processing of an MDR1 retrovirus elevates expression of multidrug resistance in primary human hematopoietic cells.

Knipper R, Kuehlcke K, Schiedlmeier B, Hildinger M, Lindemann C, Schilz AJ, Fauser AA, Fruehauf S, Zeller WJ, Ostertag W, Eckert HG, Baum C.

Gene Ther. 2001 Feb;8(3):239-46.


Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors.

Schambach A, Wodrich H, Hildinger M, Bohne J, Kräusslich HG, Baum C.

Mol Ther. 2000 Nov;2(5):435-45.