Vectorology / Biosafety

Further publications:   Gene Therapy     Regenerative Medicine     Hematopoiesis

2022

• Adeno-associated virus type 2 (AAV2) uncoating is a stepwise process and is linked to structural reorganization of the nucleolus.
  Sutter SO, Lkharrazi A, Schraner EM, Michaelsen K, Meier AF, Marx J, Vogt B, Büning H, Fraefel C.
  PLoS Pathog 2022;18(7):e1010187
  DOI: 10.1371/journal.ppat.1010187
   
• Type I Interferon Signaling Controls Gammaherpesvirus Latency In Vivo.
  Schwerk J, Kemper L, Bussey KA, Lienenklaus S, Weiss S, Čičin-Šain L, Kröger A, Kalinke U, Collins CM, Speck SH, Messerle M, Wirth D, Brinkmann MM, Hauser H, Köster M.
  Pathogens 2022;11(12):1554
  DOI: 10.3390/pathogens11121554
   
• The PI3K inhibitor pictilisib and the multikinase inhibitors pazopanib and sorafenib have an impact on Rac1 level and migration of medulloblastoma in vitro.
  Schoen LF, Craveiro RB, Pietsch T, Moritz T, Troeger A, Jordans S, Dilloo D.
  J Cell Mol Med 2022;26(23):5832-5845
  DOI: 10.1111/jcmm.17604
   
• Polarization of human iPSC-derived macrophages directs their immunological response to secondary pro-inflammatory stimuli.
  Schinke M, Meyer G, Rafiei Hashtchin A, Hetzel M, Abdin SM, Wegner T, Schwarzer A, Hansen G, Schambach A, Lachmann N, Ackermann M.
  J Immunol Regen 2022;17:100061
  DOI: 10.1016/j.regen.2022.100061
   
• AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes.
  Rode L, Bär C, Groß S, Rossi A, Meumann N, Viereck J, Abbas N, Xiao K, Riedel I, Gietz A, Zimmer K, Odenthal M, Büning H, Thum T
  Mol Ther 2022;30(12):3601-3618
  DOI: 10.1016/j.ymthe.2022.07.003
   
• . Immunogenicity of Novel AAV Capsids for Retinal Gene Therapy.
  Gehrke M, Diedrichs-Möhring M, Bogedein J, Büning H, Michalakis S, Wildner G.
  Cells 2022;11(12):1881
  DOI: 10.3390/cells11121881
   
• Development of genetic safety switches and their validation.
  Dahlke, Julia (Dr. rer. nat.)
  Medizinische Hochschule Hannover, 2022
   
• Podocalyxin-Like Protein 1 Regulates Pluripotency through the Cholesterol Biosynthesis Pathway.
  Chen W, Huang W, Pather SR, Chang W, Sung L, Wu H, Liao M, Lee C, Wu H, Wu C, Liao K, Lin C, Yang S, Lin H, Lai P, Ng C, Hu C, Chen I, Chuang C, Lai C, Lin P, Lee Y, Schuyler SC, Schambach A, Lu FL, Lu J.
  Adv Sci (Weinh) 2023;10(1):e2205451
  DOI: 10.1002/advs.202205451
   
• Development of new in vitro safety assays for gene therapy.
  Bastone, Antonella Lucía (PhD)
  Medizinische Hochschule Hannover, 2022

2021

• Optimized Adeno-Associated Virus Vectors for Efficient Transduction of Human Retinal Organoids.
  Völkner M, Pavlou M, Büning H, Michalakis S, Karl MO.
  Hum Gene Ther 2021;32(13-14):694-706
  DOI: 10.1089/hum.2020.321
   
• Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors.
  Macdonald J, Marx J, Büning H.
  Hum Gene Ther 2021;32(19-20):1096-1119
  DOI: 10.1089/hum.2021.169
   
• Improved alpharetrovirus-based Gag.MS2 particles for efficient and transient delivery of CRISPR-Cas9 into target cells. 
  Baron Y, Sens J, Lange L, Nassauer L, Klatt D, Hoffmann D, Kleppa MJ, Barbosa PV, Keisker M, Steinberg V, Suerth JD, Vondran FWR, Meyer J, Morgan M, Schambach A, Galla M.
  Mol Ther Nucleic Acids 2022;27:810-823
  DOI: 10.1016/j.omtn.2021.12.033
   
• Improved targeting of human CD4+ T cells by nanobody-modified AAV2 gene therapy vectors.
  Hamann MV, Beschorner N, Vu XK, Hauber I, Lange UC, Traenkle B, Kaiser PD, Foth D, Schneider C, Büning H, Rothbauer U, Hauber J.
  PLoS One 2021;16(12):e0261269
  DOI: 10.1371/journal.pone.0261269
   
• Rational Design of Single Copy Expression Cassettes in Defined Chromosomal Sites Overcomes Intraclonal Cell-to-Cell Expression Heterogeneity and Ensures Robust Antibody Production.
  Gödecke N, Herrmann S, Hauser H, Mayer-Bartschmid A, Trautwein M, Wirth D.
  ACS Synth Biol 2021;10(1):145-157
  DOI: 10.1021/acssynbio.0c00519
   
• 3D culture conditions support Kaposi’s sarcoma herpesvirus (KSHV) maintenance and viral spread in endothelial cells
  Dubich T, Dittrich A, Bousset K, Geffers R, Büsche G, Köster M, Hauser H, Schulz TF, Wirth D.
  J Mol Med (Berl) 2021;99(3):425-438
  DOI: 10.1007/s00109-020-02020-8
   
• Efficient Genetic Safety Switches for Future Application of iPSC-Derived Cell Transplants.
  Dahlke J, Schott JW, Vollmer Barbosa P, Klatt D, Selich A, Lachmann N, Morgan M, Moritz T, Schambach A.
  J Pers Med 2021;11(6):565
  DOI: 10.3390/jpm11060565

2020

• Design and Characterization of an “All-in-One” Lentiviral Vector System Combining Constitutive Anti-GD2 CAR Expression and Inducible Cytokines
  Katharina Zimmermann, Johannes Kuehle, Anna Christina Dragon, Melanie Galla,
  Christina Kloth, Loreen Sophie Rudek, I. Erol Sandalcioglu, Belal Neyazi, Thomas Moritz,
  Johann Meyer, Claudia Rossig, Bianca Altvater, Britta Eiz-Vesper,
  Michael Alexander Morgan, Hinrich Abken and Axel Schambach
  Cancers (Basel) 2020 Feb 6;12(2):375. doi: 10.3390/cancers12020375.
   
• Intrinsic Differential Scanning Fluorimetry for Fast and Easy Identification of Adeno-Associated Virus Serotypes
  Ruth Rieser, Magalie Penaud-Budloo, Mohammed Bouzelha, Axel Rossi, Tim Menzen, Martin Biel, Hildegard Büning, Eduard Ayuso,
  Gerhard Winter, Stylianos Michalakis
  J Pharm Sci. 2020 Jan;109(1):854-862.doi: 10.1016/j.xphs.2019.10.031.
   

• Liver-expressed Cd302 and Cr1l limit hepatitis C virus cross-species transmission to mice
  Richard J P Brown, Birthe Tegtmeyer, Julie Sheldon, Tanvi Khera, Anggakusuma, Daniel Todt, Gabrielle Vieyres, Romy Weller, Sebastian Joecks, Yudi Zhang, Svenja Sake, Dorothea Bankwitz, Kathrin Welsch, Corinne Ginkel, Michael Engelmann, Gisa Gerold, Eike Steinmann, Qinggong Yuan, Michael Ott, Florian W R Vondran , Thomas Krey, Luisa J Ströh, Csaba Miskey, Zoltán Ivics, Vanessa Herder, Wolfgang Baumgärtner, Chris Lauber, Michael Seifert, Alexander W Tarr, C Patrick McClure, Glenn Randall, Yasmine Baktash, Alexander Ploss, Viet Loan Dao Thi, Eleftherios Michailidis, Mohsan Saeed, Lieven Verhoye, Philip Meuleman, Natascha Goedecke, Dagmar Wirth, Charles M Rice, Thomas Pietschmann
  Sci Adv. 2020 Nov 4;6(45):eabd3233.doi: 10.1126/sciadv.abd3233. Print 2020 Nov.
   

• AAV Entry: Filling in the Blanks
  Hildegard Büning
  Molecular Therapy Vol. 28 No 2 February 2020 https://doi.org/10.1016/j.ymthe.2020.01.015
   

• Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cell
  Denise Klatt, Erica Cheng, Dirk Hoffmann, Giorgia Santilli, Adrian J Thrasher, Christian Brendel, Axel Schambach
  Hum Gene Ther. 2020 Feb;31(3-4):199-210.doi: 10.1089/hum.2019.194. Epub 2020 Jan 23.
   

• Ex Vivo/In vivo Gene Editing in Hepatocytes Using "All-in-One" CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template
  Simon Alexander Krooss, Zhen Dai, Florian Schmidt, Alice Rovai, Julia Fakhiri, Akshay Dhingra, Qinggong Yuan, Taihua Yang, Asha Balakrishnan, Lars Steinbrück, Sangar Srivaratharajan, Michael Peter Manns, Axel Schambach, Dirk Grimm, Jens Bohne, Amar Deep Sharma, Hildegard Büning, Michael Ott
  iScience. 2020 Jan 24;23(1):100764.doi: 10.1016/j.isci.2019.100764. Epub 2019 Dec 12.
   

• Targeted Integration of Inducible Caspase-9 in Human iPSCs Allows Efficient in vitro Clearance of iPSCs and iPSC-Macrophages
  Alexandra Lipus, Ewa Janosz, Mania Ackermann, Miriam Hetzel, Julia Dahlke, Theresa Buchegger, Stephanie Wunderlich, Ulrich Martin, Toni Cathomen, Axel Schambach, Thomas Moritz, Nico Lachmann
  Int J Mol Sci. 2020 Apr 3;21(7):2481.doi: 10.3390/ijms21072481.

2019

• Long-Term Safety and Efficacy of Gene-Pulmonary Macrophage Transplantation Therapy of PAP in Csf2ra-/-Mice
  Paritha Arumugam, Takuji Suzuki, Kenjiro Shima, Cormac McCarthy, Anthony Sallese, Matthew Wessendarp, Yan Ma, Johann Meyer, Diane Black, Claudia Chalk, Brenna Carey,Nico Lachmann, Thomas Moritz, and Bruce C. Trapnell
  Mol Ther 2019 Sep 4;27(9):1597-1611.doi: 10.1016/j.ymthe.2019.06.010. Epub 2019 Jul 2.
   
• Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
  Hildegard Büning and Arun Srivastava
  Molecular Therapy: Methods & Clinical Development Vol. 12 March 2019
   
• Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
  Sabine Charrier, Chantal Lagresle-Peyrou, Valentina Poletti, Michael Rothe, Grégory Cédrone,
  Bernard Gjata, Fulvio Mavilio, Alain Fischer, Axel Schambach, Jean-Pierre de Villartay, Marina Cavazzana, Salima Hacein-Bey-Abina and Anne Galy
  Molecular Therapy: Methods & Clinical Development Vol. 15 December 2019 doi:https://doi.org/10.1016/j.omtm.2019.08.014
   
• GluA4-Targeted AAV Vectors Deliver Genes Selectively to Interneurons while Relying on the AAV Receptor for Entry
  Jessica Hartmann, Frederic B. Thalheimer, Felix Höpfner, Thomas Kerzel, Konstantin Khodosevich, Diego García-González, Hannah Monyer, Ilka Diester, 
  Hildegard Büning, Jan E. Carette, Pascal Fries and Christian J. Buchholz
  Molecular Therapy: Methods & Clinical Development Vol. 14 September 2019, DOI: doi.org/10.1016/j.omtm.2019.07.004
   
• Model-based analysis of influenza A virus replication in genetically engineered cell lines elucidates the impact of host cell factors on key kinetic parameters of virus growth
  Tanja Laske, Mandy Bachmann, Melanie Dostert, Alexander Karlas,
  Dagmar Wirth, Timo Frensing, Thomas F. Meyer, Hansjörg Hauser, Udo Reichl
  PLoS Comput Biol. 2019 Apr 11;15(4):e1006944.doi: 10.1371/journal.pcbi.1006944.
   
• Rational Design of Gene Therapy Vectors
  Dao Pan, Hildegard Büning, Chen Ling
  Molecular Therapy: Methods & Clinical Development Vol. 12 March 2019 https://doi.org/10.1016/j.omtm.2019.01.009
   
• Vector uncoating limits adeno-associated viral vector-mediated transduction of human dendritic cells and vector immunogenicity
  Axel Rossi, Léa Dupaty, Ludovic Aillot, Liang Zhang,Célia Gallien, Michael Hallek, Margarete Odenthal, Sahil Adriouch, Anna Salvetti, Hildegard Büning
  Sci Rep. 2019 Mar 6;9(1):3631.doi: 10.1038/s41598-019-40071-1.
   
• Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application
  Juliane W. Schott, Diego León-Rico, Carolina B. Ferreira, Karen F. Buckland, Giorgia Santilli,
  Myriam A. Armant, Axel Schambach, Alessia Cavazza and Adrian J. Thrasher
  Mol Ther Methods Clin Dev. 2019 Jun 7;14:134-147.doi: 10.1016/j.omtm.2019.05.015.

2018

• Characterization of AAV vector particle stability at the single-capsid level.
  Bernaud J, Rossi A, Fis A, Gardette L, Aillot L, Büning H, Castelnovo M, Salvetti A, Faivre-Moskalenko C.
  J Biol Phys. 2018 Jun;44(2):181-194. doi: 10.1007/s10867-018-9488-5.
   
• DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs.
  Park HH, Triboulet R, Bentler M, Guda S, Du P, Xu H, Gregory RI, Brendel C, Williams DA.
  Mol Ther Nucleic Acids. 2018 Sep 7;12:591-599. doi: 10.1016/j.omtn.2018.07.002.

• Fluorescent genetic barcoding for cellular multiplex analyses.
  Maetzig T, Morgan M, Schambach A.
  Exp Hematol. 2018 Nov;67:10-17. doi: 10.1016/j.exphem.2018.08.001.

• Pooled Generation of Lentiviral Tetracycline-Regulated microRNA Embedded Short Hairpin RNA Libraries.
  Adams FF, Hoffmann T, Zuber J, Heckl D, Schambach A, Schwarzer A.
  Hum Gene Ther Methods. 2018 Feb;29(1):16-29. doi: 10.1089/hgtb.2017.182.

• 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. eCollection 2016.

• Transient Retrovirus-Based CRISPR/Cas9 All-in-One Particles for Efficient, Targeted Gene Knockout.
  Knopp Y, Geis FK, Heckl D, Horn S, Neumann T, Kuehle J, Meyer J, Fehse B, Baum C, Morgan M, Meyer J, Schambach A, Galla M.
  Mol Ther Nucleic Acids. 2018 Dec 7;13:256-274. doi: 10.1016/j.omtn.2018.09.006.

2017

• An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries.
  Adams FF, Heckl D, Hoffmann T, Talbot SR, Kloos A, Thol F, Heuser M, Zuber J, Schambach A, Schwarzer A.
  Biomaterials 2017;139:102-115
   
• Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy.
  Biasco L, Rothe M, Büning H, Schambach A.
  Mol Ther Methods Clin Dev 2017;8:21-30
   
• Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis.
  Biasco L, Rothe M, Schott JW, Schambach A.
  Hematol Oncol Clin North Am 2017;31(5):737-752
   
• Cell Cycle-Dependent Expression of Adeno-Associated Virus 2 (AAV2) Rep in Coinfections with Herpes Simplex Virus 1 (HSV-1) Gives Rise to a Mosaic of Cells Replicating either AAV2 or HSV-1.
  Franzoso FD, Seyffert M, Vogel R, Yakimovich A, de Andrade Pereira B, Meier AF, Sutter SO, Tobler K, Vogt B, Greber UF, Büning H, Ackermann M, Fraefel C.
  J Virol 2017;91(15):DOI: 10.1128/JVI.00357
   
• Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells.
  Geis FK, Galla M, Hoffmann D, Kuehle J, Zychlinski D, Maetzig T, Schott JW, Schwarzer A, Goffinet C, Goff SP, Schambach A.
  Retrovirology 2017;14(1):34
   
• Controlled re-activation of epigenetically silenced Tet promoter-driven transgene expression by targeted demethylation.
  Gödecke N, Zha L, Spencer S, Behme S, Riemer P, Rehli M, Hauser H, Wirth D.
  Nucleic Acids Res 2017;45(16):e147
   
• Small But Increasingly Mighty: Latest Advances in AAV Vector Research, Design, and Evolution.
  Grimm D, Büning H.
  Hum Gene Ther 2017;28(11):1075-1086
   
• S/MAR Element Facilitates Episomal Long-Term Persistence of Adeno-Associated Virus Vector Genomes in Proliferating Cells.
  Hagedorn C, Schnödt-Fuchs M, Boehme P, Abdelrazik H, Lipps HJ, Büning H.
  Hum Gene Ther 2017;28(12):1169-1179
   
• Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency.
  Hetzel M, Suzuki T, Hashtchin AR, Arumugam P, Carey B, Schwabbauer M, Kuhn A, Meyer J, Schambach A, Van Der Loo J, Moritz T, Trapnell BC, Lachmann N.
  Hum Gene Ther Methods 2017;28(6):318-329
   
• Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells.
  Hoffmann D, Schott JW, Geis FK, Lange L, Müller FJ, Lenz D, Zychlinski D, Steinemann D, Morgan M, Moritz T, Schambach A.
  Gene Ther 2017;24(5):298-307
   
• Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors.
  Hösel M, Huber A, Bohlen S, Lucifora J, Ronzitti G, Puzzo F, Boisgerault F, Hacker UT, Kwanten WJ, Klöting N, Blüher M, Gluschko A, Schramm M, Utermöhlen O, Bloch W, Mingozzi F, Krut O, Büning H.
  Hepatology 2017;66(1):252-265
   
• Hepatitis B Virus Activates Signal Transducer and Activator of Transcription 3 Supporting Hepatocyte Survival and Virus Replication.
  Hösel M, Quasdorff M, Ringelhan M, Kashkar H, Debey-Pascher S, Sprinzl MF, Bockmann JH, Arzberger S, Webb D, von Olshausen G, Weber A, Schultze JL, Büning H, Heikenwalder M, Protzer U.
  Cell Mol Gastroenterol Hepatol 2017;4(3):339-363
   
• The CpG-sites of the CBX3 ubiquitous chromatin opening element are critical structural determinants for the anti-silencing function.
  Kunkiel J, Gödecke N, Ackermann M, Hoffmann D, Schambach A, Lachmann N, Wirth D, Moritz T.
  Sci Rep 2017;7(1):7919
   
• Refined sgRNA efficacy prediction improves large- and small-scale CRISPR-Cas9 applications.
  Labuhn M, Adams FF, Ng M, Knoess S, Schambach A, Charpentier EM, Schwarzer A, Mateo JL, Klusmann JH, Heckl D.
  Nucleic Acids Res 2017:DOI: 10.1093/nar/gkx1268
   
• A Lentiviral Fluorescent Genetic Barcoding System for Flow Cytometry-Based Multiplex Tracking.
  Maetzig T, Ruschmann J, Lai CK, Ngom M, Imren S, Rosten P, Norddahl GL, von Krosigk N, Sanchez Milde L, May C, Selich A, Rothe M, Dhillon I, Schambach A, Humphries RK.
  Mol Ther 2017;25(3):606-620
   
• Lentiviral Fluorescent Genetic Barcoding for Multiplex Fate Tracking of Leukemic Cells.
  Maetzig T, Ruschmann J, Sanchez Milde L, Lai CK, von Krosigk N, Humphries RK.
  Mol Ther Methods Clin Dev 2017;6:54-65
   
• Improving the Quality of Adeno-Associated Viral Vector Preparations: The Challenge of Product-Related Impurities.
  Schnödt M, Büning H.
  Hum Gene Ther Methods 2017;28(3):101-108
   
• Novel Mutant AAV2 Rep Proteins Support AAV2 Replication without Blocking HSV-1 Helpervirus Replication.
  Seyffert M, Glauser DL, Schraner EM, de Oliveira AP, Mansilla-Soto J, Vogt B, Büning H, Linden RM, Ackermann M, Fraefel C.
  PLoS One 2017;12(1):e0170908
   
• Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration.
  Stahlhut M, Schambach A, Kustikova OS.
  Hum Gene Ther Methods 2017;28(4):191-204
   
• Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.
  Vink CA, Counsell JR, Perocheau DP, Karda R, Buckley SMK, Brugman MH, Galla M, Schambach A, McKay TR, Waddington SN, Howe SJ.
  Mol Ther 2017;25(8):1790-1804

2016

• 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.

2015

• 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.

2014

• 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.

2013

• 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.

2012

• 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.

2011

• 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.

2000 - 2010

• 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.

• 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.