Regenerative Medicine

Further publications:   Gene Therapy     Vectorology / Biosafety     Hematopoiesis

2019

2018

• A patient-specific induced pluripotent stem cell model for West syndrome caused by ST3GAL3 deficiency.
  van Diepen L, Buettner FFR, Hoffmann D, Thiesler CT, von Bohlen Und Halbach O, von Bohlen Und Halbach V, Jensen LR, Steinemann D, Edvardson S, Elpeleg O, Schambach A, Gerardy-Schahn R, Kuss AW.
  Eur J Hum Genet. 2018 Dec;26(12):1773-1783. doi: 10.1038/s41431-018-0220-5.
   
• An immune cell spray (ICS) formulation allows for the delivery of functional monocyte/macrophages.
  Beneke V, Küster F, Neehus AL, Hesse C, Lopez-Rodriguez E, Haake K, Rafiei Hashtchin A, Schott JW, Walter D, Braun A, Wolkers WF, Ackermann M, Lachmann N.
  Sci Rep. 2018 Nov 2;8(1):16281. doi: 10.1038/s41598-018-34524-2.
   
• Bioreactor-based mass production of human iPSC-derived macrophages enables immunotherapies against bacterial airway infections.
  Ackermann M, Kempf H, Hetzel M, Hesse C, Hashtchin AR, Brinkert K, Schott JW, Haake K, Kühnel MP, Glage S, Figueiredo C, Jonigk D, Sewald K, Schambach A, Wronski S, Moritz T, Martin U, Zweigerdt R, Munder A, Lachmann N.
  Nat Commun. 2018 Nov 30;9(1):5088. doi: 10.1038/s41467-018-07570-7.
   
• Concise Review: Towards the Clinical Translation of Induced Pluripotent Stem Cell-Derived Blood Cells-Ready for Take-Off.
  Haake K, Ackermann M, Lachmann N.
  Stem Cells Transl Med. 2019 Apr;8(4):332-339. doi: 10.1002/sctm.18-0134.
   
• Expansion of functional personalized cells with specific transgene combinations.
  Lipps C, Klein F, Wahlicht T, Seiffert V, Butueva M, Zauers J, Truschel T, Luckner M, Köster M, MacLeod R, Pezoldt J, Hühn J, Yuan Q, Müller PP, Kempf H, Zweigerdt R, Dittrich-Breiholz O, Pufe T, Beckmann R, Drescher W, Riancho J, Sañudo C, Korff T, Opalka B, Rebmann V, Göthert JR, Alves PM, Ott M, Schucht R, Hauser H, Wirth D, May T.
  Nat Commun. 2018 Mar 8;9(1):994. doi: 10.1038/s41467-018-03408-4.
   
• Human monocyte-derived macrophages inhibit HCMV spread independent of classical antiviral cytokines.
  Becker J, Kinast V, Döring M, Lipps C, Duran V, Spanier J, Tegtmeyer PK, Wirth D, Cicin-Sain L, Alcamí A, Kalinke U.
  Virulence. 2018;9(1):1669-1684. doi: 10.1080/21505594.2018.1535785.
   
• Human Teratoma-Derived Hematopoiesis Is a Highly Polyclonal Process Supported by Human Umbilical Vein Endothelial Cells.
  Philipp F, Selich A, Rothe M, Hoffmann D, Rittinghausen S, Morgan MA, Klatt D, Glage S, Lienenklaus S, Neuhaus V, Sewald K, Braun A, Schambach A.
  Stem Cell Reports. 2018 Nov 13;11(5):1051-1060. doi: 10.1016/j.stemcr.2018.09.010.
   
• iPSC-Derived Macrophages Effectively Treat Pulmonary Alveolar Proteinosis in Csf2rb-Deficient Mice.
  Mucci A, Lopez-Rodriguez E, Hetzel M, Liu S, Suzuki T, Happle C, Ackermann M, Kempf H, Hillje R, Kunkiel J, Janosz E, Brennig S, Glage S, Bankstahl JP, Dettmer S, Rodt T, Gohring G, Trapnell B, Hansen G, Trapnell C, Knudsen L, Lachmann N, Moritz T.
  Stem Cell Reports. 2018 Sep 11;11(3):696-710. doi: 10.1016/j.stemcr.2018.07.006.
   
• Murine alveolar epithelial cells and their lentivirus-mediated immortalisation.
  Sapich S, Hittinger M, Hendrix-Jastrzebski R, Repnik U, Griffiths G, May T, Wirth D, Bals R, Schneider-Daum N, Lehr CM.
  Altern Lab Anim. 2018 May;46(2):73-89.

2017

• Ex vivo Generation of Genetically Modified Macrophages from Human Induced Pluripotent Stem Cells.
  Ackermann M, Kuhn A, Kunkiel J, Merkert S, Martin U, Moritz T, Lachmann N
  Transfus Med Hemother. 2017 Jun;44(3):135-142. doi: 10.1159/000477129.

• Rapid and efficient generation of oligodendrocytes from human induced pluripotent stem cells using transcription factors.
  Ehrlich M, Mozafari S, Glatza M, Starost L, Velychko S, Hallmann AL, Cui QL, Schambach A, Kim KP, Bachelin C, Marteyn A, Hargus G, Johnson RM, Antel J, Sterneckert J, Zaehres H, Scholer HR, Baron-Van Evercooren A, Kuhlmann T.
  Proc Natl Acad Sci U S A 2017;114(11):E2243-E2252

• 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

• 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

• TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis.
  Kuhn A, Ackermann M, Mussolino C, Cathomen T, Lachmann N, Moritz T.
  Sci Rep 2017;7(1):15195

• Forced expression of human macrophage colony-stimulating factor in CD34(+) cells promotes monocyte differentiation in vitro and in vivo but blunts osteoclastogenesis in vitro.
  Montano Almendras CP, Thudium CS, Löfvall H, Moscatelli I, Schambach A, Henriksen K, Richter J.
  Eur J Haematol 2017;98(5):517-526

• Impaired IFNgamma-Signaling and Mycobacterial Clearance in IFNgammaR1-Deficient Human iPSC-Derived Macrophages.
  Neehus AL, Lam J, Haake K, Merkert S, Schmidt N, Mucci A, Ackermann M, Schubert M, Happle C, Kühnel MP, Blank P, Philipp F, Goethe R, Jonigk D, Martin U, Kalinke U, Baumann U, Schambach A, Roesler J, Lachmann N.
  Stem Cell Reports 2017:DOI:10.1016/j.stemcr.2017.11.011

• Inhibition of miRNA-212/132 improves the reprogramming of fibroblasts into induced pluripotent stem cells by de-repressing important epigenetic remodelling factors.
  Pfaff N, Liebhaber S, Möbus S, Beh-Pajooh A, Fiedler J, Pfanne A, Schambach A, Thum T, Cantz T, Moritz T.
  Stem Cell Res 2017;20:70-75

• Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells.
  Pittermann E, Lachmann N, MacLean G, Emmrich S, Ackermann M, Göhring G, Schlegelberger B, Welte K, Schambach A, Heckl D, Orkin SH, Cantz T, Klusmann JH.
  Blood Adv 2017;1(14):903-914

2016

• Massive Clonal Selection and Transiently Contributing Clones During Expansion of Mesenchymal Stem Cell Cultures Revealed by Lentiviral RGB-Barcode Technology.
  Selich A, Daudert J, Hass R, Philipp F, von Kaisenberg C, Paul G, Cornils K, Fehse B, Rittinghausen S, Schambach A, Rothe M.
  Stem Cells Transl Med. 2016 May;5(5):591-601. doi: 10.5966/sctm.2015-0176.

• Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency.
  Mucci A, Kunkiel J, Suzuki T, Brennig S, Glage S, Kühnel MP, Ackermann M, Happle C, Kuhn A, Schambach A, Trapnell BC, Hansen G, Moritz T, Lachmann N.
  Stem Cell Reports. 2016 Aug 9;7(2):292-305. doi: 10.1016/j.stemcr.2016.06.011.

• Glycomic Characterization of Induced Pluripotent Stem Cells Derived from a Patient Suffering from Phosphomannomutase 2 Congenital Disorder of Glycosylation (PMM2-CDG).
  Thiesler CT, Cajic S, Hoffmann D, Thiel C, van Diepen L, Hennig R, Sgodda M, Weiβmann R, Reichl U, Steinemann D, Diekmann U, Huber NM, Oberbeck A, Cantz T, Kuss AW, Körner C, Schambach A, Rapp E, Buettner FF.
  Mol Cell Proteomics. 2016 Apr;15(4):1435-52. doi: 10.1074/mcp.M115.054122.

• MicroRNA-Based Therapy of GATA2-Deficient Vascular Disease.
  Hartmann D, Fiedler J, Sonnenschein K, Just A, Pfanne A, Zimmer K, Remke J, Foinquinos A, Butzlaff M, Schimmel K, Maegdefessel L, Hilfiker-Kleiner D, Lachmann N, Schober A, Froese N, Heineke J, Bauersachs J, Batkai S, Thum T.
  Circulation. 2016 Dec 13;134(24):1973-1990.

• Biphasic modulation of Wnt signaling supports efficient foregut endoderm formation from human pluripotent stem cells.
  Hoepfner J, Kleinsorge M, Papp O, Ackermann M, Alfken S, Rinas U, Solodenko W, Kirschning A, Sgodda M, Cantz T.
  Cell Biol Int. 2016 May;40(5):534-48. doi: 10.1002/cbin.10590.

• Lung surfactant metabolism: early in life, early in disease and target in cell therapy.
  Lopez-Rodriguez E, Gay-Jordi G, Mucci A, Lachmann N, Serrano-Mollar A.
  Cell Tissue Res. 2016 Oct 25. [Epub ahead of print] Review.

• Long noncoding RNA Chast promotes cardiac remodeling.
  Viereck J, Kumarswamy R, Foinquinos A, Xiao K, Avramopoulos P, Kunz M, Dittrich M, Maetzig T, Zimmer K, Remke J, Just A, Fendrich J, Scherf K, Bolesani E, Schambach A, Weidemann F, Zweigerdt R, de Windt LJ, Engelhardt S, Dandekar T, Batkai S, Thum T.
  Sci Transl Med. 2016 Feb 17;8(326):326ra22. doi: 10.1126/scitranslmed.aaf1475.

• Direct Reprogramming of Hepatic Myofibroblasts into Hepatocytes In Vivo Attenuates Liver Fibrosis.
  Song G, Pacher M, Balakrishnan A, Yuan Q, Tsay HC, Yang D, Reetz J, Brandes S, Dai Z, Pützer BM, Araúzo-Bravo MJ, Steinemann D, Luedde T, Schwabe RF, Manns MP, Schöler HR, Schambach A, Cantz T, Ott M, Sharma AD.
  Cell Stem Cell. 2016 Jun 2;18(6):797-808. doi: 10.1016/j.stem.2016.01.010.

• Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase.
  Eggenschwiler R, Moslem M, Fráguas MS, Galla M, Papp O, Naujock M, Fonfara I, Gensch I, Wähner A, Beh-Pajooh A, Mussolino C, Tauscher M, Steinemann D, Wegner F, Petri S, Schambach A, Charpentier E, Cathomen T, Cantz T.
  Sci Rep. 2016 Dec 2;6:38198. doi: 10.1038/srep38198.

• Prevention of allograft rejection by use of regulatory T cells with a MHC-specific chimeric antigen receptor.
  Noyan F, Zimmermann K, Hardtke-Wolenski M, Knoefel A, Schulde E, Geffers R, Hust M, Huehn J, Galla M, Morgan M, Jokuszies A, Manns MP, Jaeckel E.
  Am J Transplant. 2016 Dec 20. doi: 10.1111/ajt.14175. [Epub ahead of print]

• Generation of HLA-universal iPSCs-derived megakaryocytes and platelets for survival under refractoriness conditions.
  Börger AK, Eicke D, Wolf C, Gras C, Aufderbeck S, Schulze K, Engels L, Eiz-Vesper B, Schambach A, Guzman CA, Lachmann N, Moritz T, Martin U, Blasczyk R, Figueiredo C.
  Mol Med. 2016 May 16;22. doi: 10.2119/molmed.2015.00235. [Epub ahead of print]

2015

• Production of mature healthy hematopoietic cells from induced pluripotent stem cells derived from an AML diagnostic sample containing the t(8;21) translocation.
  Hoffmann D, Göhring G, Heuser M, Ganser A, Schambach A, Morgan MA.
  Stem Cells. 2015 Sep 21. doi: 10.1002/stem.2207. [Epub ahead of print]

• Large-scale hematopoietic differentiation of human induced pluripotent stem cells provides granulocytes or macrophages for cell replacement therapies.
  Lachmann N, Ackermann M, Frenzel E, Liebhaber S, Brennig S, Happle C, Hoffmann D, Klimenkova O, Lüttge D, Buchegger T, Kühnel MP, Schambach A, Janciauskiene S, Figueiredo C, Hansen G, Skokowa J, Moritz T.
  Stem Cell Reports. 2015 Feb 10;4(2):282-96. doi: 10.1016/j.stemcr.2015.01.005.

2014

• FAS-based cell depletion facilitates the selective isolation of mouse induced pluripotent stem cells.
  Warlich E, Schambach A, Lock D, Wedekind D, Glage S, Eckardt D, Bosio A, Knöbel S.
  PLoS One. 2014 Jul 16;9(7):e102171. doi: 10.1371/journal.pone.0102171. eCollection 2014.

• Differential role of nonhomologous end joining factors in the generation, DNA damage response, and myeloid differentiation of human induced pluripotent stem cells.
  Felgentreff K, Du L, Weinacht KG, Dobbs K, Bartish M, Giliani S, Schlaeger T, DeVine A, Schambach A, Woodbine LJ, Davies G, Baxi SN, van der Burg M, Bleesing J, Gennery A, Manis J, Pan-Hammarström Q, Notarangelo LD.
  Proc Natl Acad Sci U S A. 2014 Jun 17;111(24):8889-94. doi: 10.1073/pnas.1323649111. Epub 2014 Jun 2.

• Promoter and lineage independent anti-silencing activity of the A2 ubiquitous chromatin opening element for optimized human pluripotent stem cell-based gene therapy.
  Ackermann M, Lachmann N, Hartung S, Eggenschwiler R, Pfaff N, Happle C, Mucci A, Göhring G, Niemann H, Hansen G, Schambach A, Cantz T, Zweigerdt R, Moritz T.
  Biomaterials. 2014 Feb;35(5):1531-42. doi: 10.1016/j.biomaterials.2013.11.024. Epub 2013 Nov 26.

2013

• Toward position-independent retroviral vector expression in pluripotent stem cells.
  Schambach A, Moritz T.
  Mol Ther. 2013 Aug;21(8):1474-7. doi: 10.1038/mt.2013.161. No abstract available.

• A ubiquitous chromatin opening element prevents transgene silencing in pluripotent stem cells and their differentiated progeny.
  Pfaff N, Lachmann N, Ackermann M, Kohlscheen S, Brendel C, Maetzig T, Niemann H, Antoniou MN, Grez M, Schambach A, Cantz T, Moritz T.
  Stem Cells. 2013 Mar;31(3):488-99. doi: 10.1002/stem.1316.

2012

• Efficient hematopoietic redifferentiation of induced pluripotent stem cells derived from primitive murine bone marrow cells.
  Pfaff N, Lachmann N, Kohlscheen S, Sgodda M, Araúzo-Bravo MJ, Greber B, Kues W, Glage S, Baum C, Niemann H, Schambach A, Cantz T, Moritz T.
  Stem Cells Dev. 2012 Mar 20;21(5):689-701. doi: 10.1089/scd.2011.0010. Epub 2011 Aug 24.

2011

• miRNA screening reveals a new miRNA family stimulating iPS cell generation via regulation of Meox2.
  Pfaff N, Fiedler J, Holzmann A, Schambach A, Moritz T, Cantz T, Thum T.
  EMBO Rep. 2011 Oct 28;12(11):1153-9. doi: 10.1038/embor.2011.176.

• Optimal reprogramming factor stoichiometry increases colony numbers and affects molecular characteristics of murine induced pluripotent stem cells.
  Tiemann U, Sgodda M, Warlich E, Ballmaier M, Schöler HR, Schambach A, Cantz T.
  Cytometry A. 2011 Jun;79(6):426-35. doi: 10.1002/cyto.a.21072. Epub 2011 May 4.

• Lentiviral vector design and imaging approaches to visualize the early stages of cellular reprogramming.
  Warlich E, Kuehle J, Cantz T, Brugman MH, Maetzig T, Galla M, Filipczyk AA, Halle S, Klump H, Schöler HR, Baum C, Schroeder T, Schambach A.
  Mol Ther. 2011 Apr;19(4):782-9. doi: 10.1038/mt.2010.314. Epub 2011 Feb 1.

2007 - 2010

• Generation and genetic modification of induced pluripotent stem cells.
  Schambach A, Cantz T, Baum C, Cathomen T.
  Expert Opin Biol Ther. 2010 Jul;10(7):1089-103. doi: 10.1517/14712598.2010.496775. Review.

• Zinc-finger nucleases meet iPS cells: Zinc positive: tailored genome engineering meets reprogramming.
  Cathomen T, Schambach A.
  Gene Ther. 2010 Jan;17(1):1-3. doi: 10.1038/gt.2009.151. Epub 2009 Nov 12. No abstract available.

• HOXB4's road map to stem cell expansion.
  Schiedlmeier B, Santos AC, Ribeiro A, Moncaut N, Lesinski D, Auer H, Kornacker K, Ostertag W, Baum C, Mallo M, Klump H.
  Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):16952-7. Epub 2007 Oct 16.