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Endothelial protein C receptor supports hematopoietic stem cell engraftment and expansion in Mpl-deficient mice.

Kohlscheen S, Schenk F, Rommel MGE, Cullmann K, Modlich U.

Blood. 2019 Mar 28;133(13):1465-1478. doi: 10.1182/blood-2018-03-837344. Epub 2019 Jan 25.


Sustained and regulated gene expression by Tet-inducible "all-in-one" retroviral vectors containing the HNRPA2B1-CBX3 UCOE®.

Cullmann K, Blokland KEC, Sebe A, Schenk F, Ivics Z, Heinz N, Modlich U.

Biomaterials. 2019 Feb;192:486-499. doi: 10.1016/j.biomaterials.2018.11.006. Epub 2018 Nov 24.


In vivo generation of human CD19-CAR T cells results in B-cell depletion and signs of cytokine release syndrome.

Pfeiffer A, Thalheimer FB, Hartmann S, Frank AM, Bender RR, Danisch S, Costa C, Wels WS, Modlich U, Stripecke R, Verhoeyen E, Buchholz CJ.

EMBO Mol Med. 2018 Nov;10(11). pii: e9158. doi: 10.15252/emmm.201809158.


Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.

Brendel C, Rothe M, Santilli G, Charrier S, Stein S, Kunkel H, Abriss D, Müller-Kuller U, Gaspar B, Modlich U, Galy A, Schambach A, Thrasher AJ, Grez M.

Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79. doi: 10.1089/humc.2017.245. Epub 2018 Apr 17.


Promises and Challenges in Hematopoietic Stem Cell Gene Therapy.

Kohlscheen S, Bonig H, Modlich U.

Hum Gene Ther. 2017 Oct;28(10):782-799. doi: 10.1089/hum.2017.141. Review.


A Cdc42/RhoA regulatory circuit downstream of glycoprotein Ib guides transendothelial platelet biogenesis.

Dütting S, Gaits-Iacovoni F, Stegner D, Popp M, Antkowiak A, van Eeuwijk JMM, Nurden P, Stritt S, Heib T, Aurbach K, Angay O, Cherpokova D, Heinz N, Baig AA, Gorelashvili MG, Gerner F, Heinze KG, Ware J, Krohne G, Ruggeri ZM, Nurden AT, Schulze H, Modlich U, Pleines I, Brakebusch C, Nieswandt B.

Nat Commun. 2017 Jun 15;8:15838. doi: 10.1038/ncomms15838.


Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy.

El Ashkar S, Van Looveren D, Schenk F, Vranckx LS, Demeulemeester J, De Rijck J, Debyser Z, Modlich U, Gijsbers R.

Mol Ther Nucleic Acids. 2017 Jun 16;7:231-245. doi: 10.1016/j.omtn.2017.04.002. Epub 2017 Apr 12.


Targeting expression to megakaryocytes and platelets by lineage-specific lentiviral vectors.

Latorre-Rey LJ, Wintterle S, Dütting S, Kohlscheen S, Abel T, Schenk F, Wingert S, Rieger MA, Nieswandt B, Heinz N, Modlich U.

J Thromb Haemost. 2017 Feb;15(2):341-355. doi: 10.1111/jth.13582.


CD30 Receptor-Targeted Lentiviral Vectors for Human Induced Pluripotent Stem Cell-Specific Gene Modification.

Friedel T, Jung-Klawitter S, Sebe A, Schenk F, Modlich U, Ivics Z, Schumann GG, Buchholz CJ, Schneider IC.

Stem Cells Dev. 2016 May 1;25(9):729-39. doi: 10.1089/scd.2015.0386. Epub 2016 Apr 11.


The heteromeric transcription factor GABP activates the ITGAM/CD11b promoter and induces myeloid differentiation.

Ripperger T, Manukjan G, Meyer J, Wolter S, Schambach A, Bohne J, Modlich U, Li Z, Skawran B, Schlegelberger B, Steinemann D.

Biochim Biophys Acta. 2015 Sep;1849(9):1145-54. doi: 10.1016/j.bbagrm.2015.07.005. Epub 2015 Jul 11.


Comparison of Different Cytokine Conditions Reveals Resveratrol as a New Molecule for Ex Vivo Cultivation of Cord Blood-Derived Hematopoietic Stem Cells.

Heinz N, Ehrnström B, Schambach A, Schwarzer A, Modlich U, Schiedlmeier B.

Stem Cells Transl Med. 2015 Sep;4(9):1064-72. doi: 10.5966/sctm.2014-0284. Epub 2015 Jul 9.


Inhibition of Thrombopoietin/Mpl Signaling in Adult Hematopoiesis Identifies New Candidates for Hematopoietic Stem Cell Maintenance.

Kohlscheen S, Wintterle S, Schwarzer A, Kamp C, Brugman MH, Breuer DC, Büsche G, Baum C, Modlich U.

PLoS One. 2015 Jul 6;10(7):e0131866. doi: 10.1371/journal.pone.0131866. eCollection 2015.


Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.

Dahl M, Doyle A, Olsson K, Månsson JE, Marques ARA, Mirzaian M, Aerts JM, Ehinger M, Rothe M, Modlich U, Schambach A, Karlsson S.

Mol Ther. 2015 May;23(5):835-844. doi: 10.1038/mt.2015.16. Epub 2015 Feb 6.


Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model.

Haemmerle R, Phaltane R, Rothe M, Schröder S, Schambach A, Moritz T, Modlich U.

Mol Ther Nucleic Acids. 2014 Oct 7;3:e200. doi: 10.1038/mtna.2014.51.


Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia.

Jaako P, Debnath S, Olsson K, Modlich U, Rothe M, Schambach A, Flygare J, Karlsson S.

Haematologica. 2014 Dec;99(12):1792-8. doi: 10.3324/haematol.2014.111195. Epub 2014 Sep 12.


Lentiviral MGMT(P140K)-mediated in vivo selection employing a ubiquitous chromatin opening element (A2UCOE) linked to a cellular promoter.

Phaltane R, Lachmann N, Brennig S, Ackermann M, Modlich U, Moritz T.

Biomaterials. 2014 Aug;35(25):7204-13. doi: 10.1016/j.biomaterials.2014.05.001. Epub 2014 May 27.


Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.

Braun CJ, Boztug K, Paruzynski A, Witzel M, Schwarzer A, Rothe M, Modlich U, Beier R, Göhring G, Steinemann D, Fronza R, Ball CR, Haemmerle R, Naundorf S, Kühlcke K, Rose M, Fraser C, Mathias L, Ferrari R, Abboud MR, Al-Herz W, Kondratenko I, Maródi L, Glimm H, Schlegelberger B, Schambach A, Albert MH, Schmidt M, von Kalle C, Klein C.

Sci Transl Med. 2014 Mar 12;6(227):227ra33. doi: 10.1126/scitranslmed.3007280.


Generation of iPSCs from genetically corrected Brca2 hypomorphic cells: implications in cell reprogramming and stem cell therapy.

Navarro S, Moleiro V, Molina-Estevez FJ, Lozano ML, Chinchon R, Almarza E, Quintana-Bustamante O, Mostoslavsky G, Maetzig T, Galla M, Heinz N, Schiedlmeier B, Torres Y, Modlich U, Samper E, Río P, Segovia JC, Raya A, Güenechea G, Izpisua-Belmonte JC, Bueren JA.

Stem Cells. 2014 Feb;32(2):436-46. doi: 10.1002/stem.1586.


Efficiency and safety of O⁶-methylguanine DNA methyltransferase (MGMT(P140K))-mediated in vivo selection in a humanized mouse model.

Phaltane R, Haemmerle R, Rothe M, Modlich U, Moritz T.

Hum Gene Ther. 2014 Feb;25(2):144-55. doi: 10.1089/hum.2013.171. Epub 2014 Jan 7.


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.


Chromosomal instability and telomere shortening in long-term culture of hematopoietic stem cells: insights from a cell culture model of RPS14 haploinsufficiency.

Thomay K, Schienke A, Vajen B, Modlich U, Schambach A, Hofmann W, Schlegelberger B, Göhring G.

Cytogenet Genome Res. 2014;142(1):14-20. doi: 10.1159/000356096. Epub 2013 Oct 26.


Mutant IDH1 promotes leukemogenesis in vivo and can be specifically targeted in human AML.

Chaturvedi A, Araujo Cruz MM, Jyotsana N, Sharma A, Yun H, Görlich K, Wichmann M, Schwarzer A, Preller M, Thol F, Meyer J, Haemmerle R, Struys EA, Jansen EE, Modlich U, Li Z, Sly LM, Geffers R, Lindner R, Manstein DJ, Lehmann U, Krauter J, Ganser A, Heuser M.

Blood. 2013 Oct 17;122(16):2877-87. doi: 10.1182/blood-2013-03-491571. Epub 2013 Aug 16.


From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.

Stein S, Scholz S, Schwäble J, Sadat MA, Modlich U, Schultze-Strasser S, Diaz M, Chen-Wichmann L, Müller-Kuller U, Brendel C, Fronza R, Kaufmann KB, Naundorf S, Pech NK, Travers JB, Matute JD, Presson RG Jr, Sandusky GE, Kunkel H, Rudolf E, Dillmann A, von Kalle C, Kühlcke K, Baum C, Schambach A, Dinauer MC, Schmidt M, Grez M.

Hum Gene Ther Clin Dev. 2013 Jun;24(2):86-98. doi: 10.1089/humc.2013.019.


Evaluating a ligation-mediated PCR and pyrosequencing method for the detection of clonal contribution in polyclonal retrovirally transduced samples.

Brugman MH, Suerth JD, Rothe M, Suerbaum S, Schambach A, Modlich U, Kustikova O, Baum C.

Hum Gene Ther Methods. 2013 Apr;24(2):68-79. doi: 10.1089/hgtb.2012.175. Epub 2013 Mar 14.


Histone methyltransferase Suv39h1 deficiency prevents Myc-induced chromosomal instability in murine myeloid leukemias.

Vajen B, Modlich U, Schienke A, Wolf S, Skawran B, Hofmann W, Büsche G, Kreipe H, Baum C, Santos-Barriopedro I, Vaquero A, Schlegelberger B, Rudolph C.

Genes Chromosomes Cancer. 2013 Apr;52(4):423-30. doi: 10.1002/gcc.22040. Epub 2013 Jan 23.


Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents.

Rittelmeyer I, Rothe M, Brugman MH, Iken M, Schambach A, Manns MP, Baum C, Modlich U, Ott M.

Hepatology. 2013 Jul;58(1):397-408. doi: 10.1002/hep.26204. Epub 2013 May 27.


Selection for Evi1 activation in myelomonocytic leukemia induced by hyperactive signaling through wild-type NRas.

Wolf S, Rudolph C, Morgan M, Büsche G, Salguero G, Stripecke R, Schlegelberger B, Baum C, Modlich U.

Oncogene. 2013 Jun 20;32(25):3028-38. doi: 10.1038/onc.2012.329. Epub 2012 Jul 30.


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.


Lentiviral vector induced insertional haploinsufficiency of Ebf1 causes murine leukemia.

Heckl D, Schwarzer A, Haemmerle R, Steinemann D, Rudolph C, Skawran B, Knoess S, Krause J, Li Z, Schlegelberger B, Baum C, Modlich U.

Mol Ther. 2012 Jun;20(6):1187-95. doi: 10.1038/mt.2012.59. Epub 2012 Apr 3.


Identity, potency, in vivo viability, and scaling up production of lentiviral vector-induced dendritic cells for melanoma immunotherapy.

Pincha M, Sundarasetty BS, Salguero G, Gutzmer R, Garritsen H, Macke L, Schneider A, Lenz D, Figueiredo C, Blasczyk R, Ruggiero E, Schmidt M, von Kalle C, Puff C, Modlich U, von der Leyen H, Wicke DC, Ganser A, Stripecke R.

Hum Gene Ther Methods. 2012 Feb;23(1):38-55. doi: 10.1089/hgtb.2011.170.


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.


MicroRNA-150-regulated vectors allow lymphocyte-sparing transgene expression in hematopoietic gene therapy.

Lachmann N, Jagielska J, Heckl D, Brennig S, Pfaff N, Maetzig T, Modlich U, Cantz T, Gentner B, Schambach A, Moritz T.

Gene Ther. 2012 Sep;19(9):915-24. doi: 10.1038/gt.2011.148. Epub 2011 Oct 6.


Concise review: managing genotoxicity in the therapeutic modification of stem cells.

Baum C, Modlich U, Göhring G, Schlegelberger B.

Stem Cells. 2011 Oct;29(10):1479-84. doi: 10.1002/stem.716. 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.


Epidermal growth factor improves lentivirus vector gene transfer into primary mouse hepatocytes.

Rothe M, Rittelmeyer I, Iken M, Rüdrich U, Schambach A, Glage S, Manns MP, Baum C, Bock M, Ott M, Modlich U.

Gene Ther. 2012 Apr;19(4):425-34. doi: 10.1038/gt.2011.117. Epub 2011 Aug 18.


CTF/NF1 transcription factors act as potent genetic insulators for integrating gene transfer vectors.

Gaussin A, Modlich U, Bauche C, Niederländer NJ, Schambach A, Duros C, Artus A, Baum C, Cohen-Haguenauer O, Mermod N.

Gene Ther. 2012 Jan;19(1):15-24. doi: 10.1038/gt.2011.70. Epub 2011 May 12.


Lentiviral vectors for induction of self-differentiation and conditional ablation of dendritic cells.

Pincha M, Salguero G, Wedekind D, Sundarasetty BS, Lin A, Kasahara N, Brugman MH, Jirmo AC, Modlich U, Gutzmer R, Büsche G, Ganser A, Stripecke R.

Gene Ther. 2011 Aug;18(8):750-64. doi: 10.1038/gt.2011.15. Epub 2011 Mar 17.


Lentiviral gene transfer regenerates hematopoietic stem cells in a mouse model for Mpl-deficient aplastic anemia.

Heckl D, Wicke DC, Brugman MH, Meyer J, Schambach A, Büsche G, Ballmaier M, Baum C, Modlich U.

Blood. 2011 Apr 7;117(14):3737-47. doi: 10.1182/blood-2010-09-308262. Epub 2011 Feb 2.


Polyclonal fluctuation of lentiviral vector-transduced and expanded murine hematopoietic stem cells.

Maetzig T, Brugman MH, Bartels S, Heinz N, Kustikova OS, Modlich U, Li Z, Galla M, Schiedlmeier B, Schambach A, Baum C.

Blood. 2011 Mar 17;117(11):3053-64. doi: 10.1182/blood-2010-08-303222. Epub 2011 Jan 19.


Cell and virus genetics at the roots of gene therapy, retrovirology, and hematopoietic stem cell biology: Wolfram Ostertag (1937-2010).

Stocking C, Grez M, Fehse B, von Laer D, Itoh K, Prassolov V, Nowock J, Kühlcke K, Just U, Schröder T, Klump H, Schiedlmeier B, Grassman E, Meyer J, Li Z, Schambach A, Modlich U, Kustikova O, Galla M, Bode J, Zander A, Baum C.

Hum Gene Ther. 2010 Nov;21(11):1501-3. doi: 10.1089/hum.2010.1901. No abstract available.


Gene therapy of MPL deficiency: challenging balance between leukemia and pancytopenia.

Wicke DC, Meyer J, Buesche G, Heckl D, Kreipe H, Li Z, Welte KH, Ballmaier M, Baum C, Modlich U.

Mol Ther. 2010 Feb;18(2):343-52. doi: 10.1038/mt.2009.233. Epub 2009 Oct 20. Erratum in: Mol Ther. 2010 Feb;18(2):448.


Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region.

Arumugam PI, Higashimoto T, Urbinati F, Modlich U, Nestheide S, Xia P, Fox C, Corsinotti A, Baum C, Malik P.

Mol Ther. 2009 Nov;17(11):1929-37. doi: 10.1038/mt.2009.183. Epub 2009 Aug 25.


Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors.

Modlich U, Navarro S, Zychlinski D, Maetzig T, Knoess S, Brugman MH, Schambach A, Charrier S, Galy A, Thrasher AJ, Bueren J, Baum C.

Mol Ther. 2009 Nov;17(11):1919-28. doi: 10.1038/mt.2009.179. Epub 2009 Aug 11.


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.


Leukemia diagnosis in murine bone marrow transplantation models.

Li Z, Modlich U, Anjali M.

Methods Mol Biol. 2009;506:311-29. doi: 10.1007/978-1-59745-409-4_21.


Murine hematopoietic stem cell transduction using retroviral vectors.

Modlich U, Schambach A, Li Z, Schiedlmeier B.

Methods Mol Biol. 2009;506:23-31. doi: 10.1007/978-1-59745-409-4_3.


Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16.

Modlich U, Schambach A, Brugman MH, Wicke DC, Knoess S, Li Z, Maetzig T, Rudolph C, Schlegelberger B, Baum C.

Leukemia. 2008 Aug;22(8):1519-28. doi: 10.1038/leu.2008.118. Epub 2008 May 22.


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.


[Gene therapy of SCID-X1].

Baum C, Schambach A, Modlich U, Thrasher A.

Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2007 Dec;50(12):1507-17. Review. German.


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