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Axel Schambach, Ph.D.

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Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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  1. Jäger B, Klatt D, Plappert L, Golpon H, Lienenklaus S, Barbosa PD, Schambach A, Prasse A. CXCR4/MIF axis amplifies tumor growth and epithelial-mesenchymal interaction in non-small cell lung cancer. Cell Signal. 2020 May 16; 109672. PMID: 32428553.
    Citations:    
  2. Lipus A, Janosz E, Ackermann M, Hetzel M, Dahlke J, Buchegger T, Wunderlich S, Martin U, Cathomen T, Schambach A, Moritz T, Lachmann N. Targeted Integration of Inducible Caspase-9 in Human iPSCs Allows Efficient in vitro Clearance of iPSCs and iPSC-Macrophages. Int J Mol Sci. 2020 Apr 03; 21(7). PMID: 32260086.
    Citations:    
  3. Garcia-Perez L, van Eggermond M, van Roon L, Vloemans SA, Cordes M, Schambach A, Rothe M, Berghuis D, Lagresle-Peyrou C, Cavazzana M, Zhang F, Thrasher AJ, Salvatori D, Meij P, Villa A, Van Dongen JJM, Zwaginga JJ, van der Burg M, Gaspar HB, Lankester A, Staal FJT, Pike-Overzet K. Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:666-682. PMID: 32322605.
    Citations:    
  4. Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A, Klatt D, Bentler M, Pellin D, Christiansen L, Schambach A, Manis J, Trebeden-Negre H, Bonner M, Esrick E, Veres G, Armant M, Williams DA. Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:589-600. PMID: 32300607.
    Citations:    
  5. Hoffmann D, Kuehle J, Lenz D, Philipp F, Zychlinski D, Lachmann N, Moritz T, Steinemann D, Morgan M, Skokowa J, Klein C, Schambach A. Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells. Gene Ther. 2020 Feb 12. PMID: 32051561.
    Citations:    
  6. Zimmermann K, Kuehle J, Dragon AC, Galla M, Kloth C, Rudek LS, Sandalcioglu IE, Neyazi B, Moritz T, Meyer J, Rossig C, Altvater B, Eiz-Vesper B, Morgan MA, Abken H, Schambach A. Design and Characterization of an "All-in-One" Lentiviral Vector System Combining Constitutive Anti-GD2 CAR Expression and Inducible Cytokines. Cancers (Basel). 2020 Feb 06; 12(2). PMID: 32041222.
    Citations:    
  7. Müller S, Bexte T, Gebel V, Kalensee F, Stolzenberg E, Hartmann J, Koehl U, Schambach A, Wels WS, Modlich U, Ullrich E. High Cytotoxic Efficiency of Lentivirally and Alpharetrovirally Engineered CD19-Specific Chimeric Antigen Receptor Natural Killer Cells Against Acute Lymphoblastic Leukemia. Front Immunol. 2019; 10:3123. PMID: 32117200.
    Citations:    
  8. Koniaeva E, Stahlhut M, Lange L, Sauer MG, Kustikova OS, Schambach A. Conditional Immortalization of Lymphoid Progenitors via Tetracycline-Regulated LMO2 Expression. Hum Gene Ther. 2020 Feb; 31(3-4):183-198. PMID: 31760808.
    Citations:    
  9. Klatt D, Cheng E, Hoffmann D, Santilli G, Thrasher AJ, Brendel C, Schambach A. Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells. Hum Gene Ther. 2020 Feb; 31(3-4):199-210. PMID: 31773990.
    Citations:    
  10. Lange L, Hoffmann D, Schwarzer A, Ha TC, Philipp F, Lenz D, Morgan M, Schambach A. Inducible Forward Programming of Human Pluripotent Stem Cells to Hemato-endothelial Progenitor Cells with Hematopoietic Progenitor Potential. Stem Cell Reports. 2020 01 14; 14(1):122-137. PMID: 31839543.
    Citations:    
  11. Krooss SA, Dai Z, Schmidt F, Rovai A, Fakhiri J, Dhingra A, Yuan Q, Yang T, Balakrishnan A, Steinbrück L, Srivaratharajan S, Manns MP, Schambach A, Grimm D, Bohne J, Sharma AD, Büning H, Ott M. Ex Vivo/In vivo Gene Editing in Hepatocytes Using "All-in-One" CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template. iScience. 2020 Jan 24; 23(1):100764. PMID: 31887661.
    Citations:    
  12. Maluski M, Ghosh A, Herbst J, Scholl V, Baumann R, Huehn J, Geffers R, Meyer J, Maul H, Eiz-Vesper B, Krueger A, Schambach A, van den Brink MR, Sauer MG. Chimeric antigen receptor-induced BCL11B suppression propagates NK-like cell development. J Clin Invest. 2019 12 02; 129(12):5108-5122. PMID: 31479431.
    Citations:    
  13. Selich A, Zimmermann K, Tenspolde M, Dittrich-Breiholz O, von Kaisenberg C, Schambach A, Rothe M. Umbilical cord as a long-term source of activatable mesenchymal stromal cells for immunomodulation. Stem Cell Res Ther. 2019 09 23; 10(1):285. PMID: 31547865.
    Citations:    
  14. Klatt D, Cheng E, Philipp F, Selich A, Dahlke J, Schmidt RE, Schott JW, Büning H, Hoffmann D, Thrasher AJ, Schambach A. Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes. Stem Cell Reports. 2019 10 08; 13(4):590-598. PMID: 31543470.
    Citations:    
  15. Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A. Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Mol Ther Methods Clin Dev. 2019 Dec 13; 15:232-245. PMID: 31720302.
    Citations:    
  16. Selich A, Ha TC, Morgan M, Falk CS, von Kaisenberg C, Schambach A, Rothe M. Cytokine Selection of MSC Clones with Different Functionality. Stem Cell Reports. 2019 08 13; 13(2):262-273. PMID: 31303506.
    Citations:    
  17. Hetzel M, Lopez-Rodriguez E, Mucci A, Nguyen AHH, Suzuki T, Shima K, Buchegger T, Dettmer S, Rodt T, Bankstahl JP, Malik P, Knudsen L, Schambach A, Hansen G, Trapnell BC, Lachmann N, Moritz T. Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis. Haematologica. 2020 Apr; 105(4):1147-1157. PMID: 31289207.
    Citations:    
  18. Löfvall H, Rothe M, Schambach A, Henriksen K, Richter J, Moscatelli I. Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice. Hum Gene Ther. 2019 11; 30(11):1395-1404. PMID: 31179768.
    Citations:    
  19. Schott JW, León-Rico D, Ferreira CB, Buckland KF, Santilli G, Armant MA, Schambach A, Cavazza A, Thrasher AJ. Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application. Mol Ther Methods Clin Dev. 2019 Sep 13; 14:134-147. PMID: 31338385.
    Citations:    
  20. Oberschmidt O, Morgan M, Huppert V, Kessler J, Gardlowski T, Matthies N, Aleksandrova K, Arseniev L, Schambach A, Koehl U, Kloess S. Development of Automated Separation, Expansion, and Quality Control Protocols for Clinical-Scale Manufacturing of Primary Human NK Cells and Alpharetroviral Chimeric Antigen Receptor Engineering. Hum Gene Ther Methods. 2019 06; 30(3):102-120. PMID: 30997855.
    Citations:    
  21. Kloess S, Oberschmidt O, Dahlke J, Vu XK, Neudoerfl C, Kloos A, Gardlowski T, Matthies N, Heuser M, Meyer J, Sauer M, Falk C, Koehl U, Schambach A, Morgan MA. Preclinical Assessment of Suitable Natural Killer Cell Sources for Chimeric Antigen Receptor Natural Killer-Based "Off-the-Shelf" Acute Myeloid Leukemia Immunotherapies. Hum Gene Ther. 2019 04; 30(4):381-401. PMID: 30734584.
    Citations:    
  22. Klapdor R, Wang S, Morgan M, Dörk T, Hacker U, Hillemanns P, Büning H, Schambach A. Characterization of a Novel Third-Generation Anti-CD24-CAR against Ovarian Cancer. Int J Mol Sci. 2019 Feb 03; 20(3). PMID: 30717444.
    Citations:    
  23. 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. Bioreactor-based mass production of human iPSC-derived macrophages enables immunotherapies against bacterial airway infections. Nat Commun. 2018 11 30; 9(1):5088. PMID: 30504915.
    Citations:    Fields:    Translation:HumansAnimalsCells
  24. Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling ßAS3 Globin for Gene Therapy for Sickle Cell Disease. Mol Ther Methods Clin Dev. 2018 Dec 14; 11:167-179. PMID: 30533448.
    Citations:    
  25. Morgan MA, Schambach A. Engineering CAR-T Cells for Improved Function Against Solid Tumors. Front Immunol. 2018; 9:2493. PMID: 30420856.
    Citations:    
  26. 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. Human Teratoma-Derived Hematopoiesis Is a Highly Polyclonal Process Supported by Human Umbilical Vein Endothelial Cells. Stem Cell Reports. 2018 11 13; 11(5):1051-1060. PMID: 30344010.
    Citations:    Fields:    
  27. Paredes R, Schneider M, Stevens A, White DJ, K Williamson AJ, Muter J, Pearson S, Kelly JR, Connors K, Wiseman DH, Chadwick JA, Löffler H, Teng HY, Lovell S, Unwin R, van de Vrugt HJ, Smith H, Kustikova O, Schambach A, P Somervaille TC, Pierce A, Whetton AD, Meyer S. Erratum: EVI1 carboxy-terminal phosphorylation is ATM-mediated and sustains transcriptional modulation and self-renewal via enhanced CtBP1 association. Nucleic Acids Res. 2018 09 19; 46(16):8663. PMID: 30102373.
    Citations:    Fields:    
  28. 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. Transient Retrovirus-Based CRISPR/Cas9 All-in-One Particles for Efficient, Targeted Gene Knockout. Mol Ther Nucleic Acids. 2018 Dec 07; 13:256-274. PMID: 30317165.
    Citations:    
  29. Paredes R, Schneider M, Stevens A, White DJ, Williamson AJK, Muter J, Pearson S, Kelly JR, Connors K, Wiseman DH, Chadwick JA, Löffler H, Teng HY, Lovell S, Unwin R, van de Vrugt HJ, Smith H, Kustikova O, Schambach A, Somervaille TCP, Pierce A, Whetton AD, Meyer S. EVI1 carboxy-terminal phosphorylation is ATM-mediated and sustains transcriptional modulation and self-renewal via enhanced CtBP1 association. Nucleic Acids Res. 2018 09 06; 46(15):7662-7674. PMID: 29939287.
    Citations:    Fields:    
  30. Morgan MA, Schambach A. Chimeric Antigen Receptor T Cells: Extending Translation from Liquid to Solid Tumors. Hum Gene Ther. 2018 10; 29(10):1083-1097. PMID: 30156435.
    Citations:    Fields:    
  31. Hammerschmidt SI, Werth K, Rothe M, Galla M, Permanyer M, Patzer GE, Bubke A, Frenk DN, Selich A, Lange L, Schambach A, Bošnjak B, Förster R. CRISPR/Cas9 Immunoengineering of Hoxb8-Immortalized Progenitor Cells for Revealing CCR7-Mediated Dendritic Cell Signaling and Migration Mechanisms in vivo. Front Immunol. 2018; 9:1949. PMID: 30210501.
    Citations:    
  32. Maetzig T, Morgan M, Schambach A. Fluorescent genetic barcoding for cellular multiplex analyses. Exp Hematol. 2018 11; 67:10-17. PMID: 30098396.
    Citations:    Fields:    
  33. 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. A patient-specific induced pluripotent stem cell model for West syndrome caused by ST3GAL3 deficiency. Eur J Hum Genet. 2018 12; 26(12):1773-1783. PMID: 30089820.
    Citations:    
  34. Salari A, Thomay K, Lentes J, Ebersold J, Hagedorn M, Skawran B, Davenport C, Schambach A, Schlegelberger B, Göhring G. Effect of TP53 contact and conformational mutations on cell survival and erythropoiesis of human hematopoietic stem cells in a long term culture model. Oncotarget. 2018 Jul 06; 9(52):29869-29876. PMID: 30042819.
    Citations:    
  35. 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. Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease. Hum Gene Ther Clin Dev. 2018 06; 29(2):69-79. PMID: 29664709.
    Citations:    Fields:    
  36. Poletti V, Charrier S, Corre G, Gjata B, Vignaud A, Zhang F, Rothe M, Schambach A, Gaspar HB, Thrasher AJ, Mavilio F. Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency. Mol Ther Methods Clin Dev. 2018 Jun 15; 9:257-269. PMID: 29707600.
    Citations:    
  37. Labuhn M, Adams FF, Ng M, Knoess S, Schambach A, Charpentier EM, Schwarzer A, Mateo JL, Klusmann JH, Heckl D. Refined sgRNA efficacy prediction improves large- and small-scale CRISPR-Cas9 applications. Nucleic Acids Res. 2018 02 16; 46(3):1375-1385. PMID: 29267886.
    Citations: 1     Fields:    
  38. Adams FF, Hoffmann T, Zuber J, Heckl D, Schambach A, Schwarzer A. Pooled Generation of Lentiviral Tetracycline-Regulated microRNA Embedded Short Hairpin RNA Libraries. Hum Gene Ther Methods. 2018 02; 29(1):16-29. PMID: 29325442.
    Citations:    Fields:    Translation:HumansCells
  39. Ghosh S, Carmo M, Calero-Garcia M, Ricciardelli I, Bustamante Ogando JC, Blundell MP, Schambach A, Ashton-Rickardt PG, Booth C, Ehl S, Lehmberg K, Thrasher AJ, Gaspar HB. T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations. J Allergy Clin Immunol. 2018 09; 142(3):904-913.e3. PMID: 29355678.
    Citations: 1     Fields:    
  40. 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. Impaired IFN?-Signaling and Mycobacterial Clearance in IFN?R1-Deficient Human iPSC-Derived Macrophages. Stem Cell Reports. 2018 01 09; 10(1):7-16. PMID: 29249666.
    Citations:    Fields:    Translation:HumansCells
  41. Hetzel M, Mucci A, Blank P, Nguyen AHH, Schiller J, Halle O, Kühnel MP, Billig S, Meineke R, Brand D, Herder V, Baumgärtner W, Bange FC, Goethe R, Jonigk D, Förster R, Gentner B, Casanova JL, Bustamante J, Schambach A, Kalinke U, Lachmann N. Hematopoietic stem cell gene therapy for IFN?R1 deficiency protects mice from mycobacterial infections. Blood. 2018 02 01; 131(5):533-545. PMID: 29233822.
    Citations: 1     Fields:    Translation:AnimalsCells
  42. Schambach A, Schott JW, Morgan MA. Uncoupling the Oncogenic Engine. Cancer Res. 2017 11 15; 77(22):6060-6064. PMID: 29097608.
    Citations:    Fields:    Translation:HumansCells
  43. Geis FK, Galla M, Hoffmann D, Kuehle J, Zychlinski D, Maetzig T, Schott JW, Schwarzer A, Goffinet C, Goff SP, Schambach A. Correction to: Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells. Retrovirology. 2017 10 18; 14(1):48. PMID: 29047401.
    Citations:    Fields:    
  44. Biasco L, Rothe M, Büning H, Schambach A. Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy. Mol Ther Methods Clin Dev. 2018 Mar 16; 8:21-30. PMID: 29159200.
    Citations: 1     
  45. Moscatelli I, Löfvall H, Schneider Thudium C, Rothe M, Montano C, Kertész Z, Sirin M, Schulz A, Schambach A, Henriksen K, Richter J. Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis. Hum Gene Ther. 2018 08; 29(8):938-949. PMID: 28726516.
    Citations:    Fields:    
  46. Biasco L, Rothe M, Schott JW, Schambach A. Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis. Hematol Oncol Clin North Am. 2017 10; 31(5):737-752. PMID: 28895844.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  47. Peking P, Koller U, Duarte B, Murillas R, Wolf S, Maetzig T, Rothe M, Kocher T, García M, Brachtl G, Schambach A, Larcher F, Reichelt J, Bauer JW, Murauer EM. An RNA-targeted therapy for dystrophic epidermolysis bullosa. Nucleic Acids Res. 2017 Sep 29; 45(17):10259-10269. PMID: 28973459.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  48. 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. Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency. Hum Gene Ther Methods. 2017 12; 28(6):318-329. PMID: 28854814.
    Citations:    Fields:    Translation:HumansAnimalsCells
  49. Klapdor R, Wang S, Hacker U, Büning H, Morgan M, Dörk T, Hillemanns P, Schambach A. Improved Killing of Ovarian Cancer Stem Cells by Combining a Novel Chimeric Antigen Receptor-Based Immunotherapy and Chemotherapy. Hum Gene Ther. 2017 10; 28(10):886-896. PMID: 28836469.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  50. Klöß S, Oberschmidt O, Morgan M, Dahlke J, Arseniev L, Huppert V, Granzin M, Gardlowski T, Matthies N, Soltenborn S, Schambach A, Koehl U. Optimization of Human NK Cell Manufacturing: Fully Automated Separation, Improved Ex Vivo Expansion Using IL-21 with Autologous Feeder Cells, and Generation of Anti-CD123-CAR-Expressing Effector Cells. Hum Gene Ther. 2017 10; 28(10):897-913. PMID: 28810809.
    Citations: 4     Fields:    Translation:HumansCells
  51. Kunkiel J, Gödecke N, Ackermann M, Hoffmann D, Schambach A, Lachmann N, Wirth D, Moritz T. The CpG-sites of the CBX3 ubiquitous chromatin opening element are critical structural determinants for the anti-silencing function. Sci Rep. 2017 08 11; 7(1):7919. PMID: 28801671.
    Citations:    Fields:    
  52. Schwarzer A, Emmrich S, Schmidt F, Beck D, Ng M, Reimer C, Adams FF, Grasedieck S, Witte D, Käbler S, Wong JWH, Shah A, Huang Y, Jammal R, Maroz A, Jongen-Lavrencic M, Schambach A, Kuchenbauer F, Pimanda JE, Reinhardt D, Heckl D, Klusmann JH. The non-coding RNA landscape of human hematopoiesis and leukemia. Nat Commun. 2017 08 09; 8(1):218. PMID: 28794406.
    Citations: 8     Fields:    Translation:HumansCells
  53. Stahlhut M, Schambach A, Kustikova OS. Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration. Hum Gene Ther Methods. 2017 08; 28(4):191-204. PMID: 28683573.
    Citations:    Fields:    Translation:AnimalsCells
  54. 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. Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells. Blood Adv. 2017 Jun 13; 1(14):903-914. PMID: 29296734.
    Citations:    
  55. Geis FK, Galla M, Hoffmann D, Kuehle J, Zychlinski D, Maetzig T, Schott JW, Schwarzer A, Goffinet C, Goff SP, Schambach A. Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells. Retrovirology. 2017 05 31; 14(1):34. PMID: 28569216.
    Citations: 2     Fields:    Translation:AnimalsCells
  56. Vink CA, Counsell JR, Perocheau DP, Karda R, Buckley SMK, Brugman MH, Galla M, Schambach A, McKay TR, Waddington SN, Howe SJ. Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy. Mol Ther. 2017 08 02; 25(8):1790-1804. PMID: 28550974.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  57. Adams FF, Heckl D, Hoffmann T, Talbot SR, Kloos A, Thol F, Heuser M, Zuber J, Schambach A, Schwarzer A. An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries. Biomaterials. 2017 Sep; 139:102-115. PMID: 28599149.
    Citations: 1     Fields:    Translation:HumansCells
  58. Huang J, Khan A, Au BC, Barber DL, López-Vásquez L, Prokopishyn NL, Boutin M, Rothe M, Rip JW, Abaoui M, Nagree MS, Dworski S, Schambach A, Keating A, West ML, Klassen J, Turner PV, Sirrs S, Rupar CA, Auray-Blais C, Foley R, Medin JA. Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease. Mol Ther Methods Clin Dev. 2017 Jun 16; 5:241-258. PMID: 28603745.
    Citations: 3     
  59. Debnath S, Jaako P, Siva K, Rothe M, Chen J, Dahl M, Gaspar HB, Flygare J, Schambach A, Karlsson S. Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia. Mol Ther. 2017 08 02; 25(8):1805-1814. PMID: 28434866.
    Citations:    Fields:    Translation:HumansAnimalsCells
  60. Westhaus S, Deest M, Nguyen ATX, Stanke F, Heckl D, Costa R, Schambach A, Manns MP, Berg T, Vondran FWR, Sarrazin C, Ciesek S, von Hahn T. Scavenger receptor class B member 1 (SCARB1) variants modulate hepatitis C virus replication cycle and viral load. J Hepatol. 2017 08; 67(2):237-245. PMID: 28363797.
    Citations: 1     Fields:    Translation:HumansCells
  61. Pfaff N, Liebhaber S, Möbus S, Beh-Pajooh A, Fiedler J, Pfanne A, Schambach A, Thum T, Cantz T, Moritz T. Inhibition of miRNA-212/132 improves the reprogramming of fibroblasts into induced pluripotent stem cells by de-repressing important epigenetic remodelling factors. Stem Cell Res. 2017 04; 20:70-75. PMID: 28314201.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  62. Montano Almendras CP, Thudium CS, Löfvall H, Moscatelli I, Schambach A, Henriksen K, Richter J. Forced expression of human macrophage colony-stimulating factor in CD34+ cells promotes monocyte differentiation in vitro and in vivo but blunts osteoclastogenesis in vitro. Eur J Haematol. 2017 May; 98(5):517-526. PMID: 28160330.
    Citations: 3     Fields:    Translation:HumansAnimalsCells
  63. 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, Schöler HR, Baron-Van Evercooren A, Kuhlmann T. Rapid and efficient generation of oligodendrocytes from human induced pluripotent stem cells using transcription factors. Proc Natl Acad Sci U S A. 2017 03 14; 114(11):E2243-E2252. PMID: 28246330.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  64. Endele M, Loeffler D, Kokkaliaris KD, Hilsenbeck O, Skylaki S, Hoppe PS, Schambach A, Stanley ER, Schroeder T. CSF-1-induced Src signaling can instruct monocytic lineage choice. Blood. 2017 03 23; 129(12):1691-1701. PMID: 28159742.
    Citations:    Fields:    Translation:AnimalsCells
  65. 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. A Lentiviral Fluorescent Genetic Barcoding System for Flow Cytometry-Based Multiplex Tracking. Mol Ther. 2017 03 01; 25(3):606-620. PMID: 28253481.
    Citations: 4     Fields:    Translation:HumansCells
  66. Müller LU, Milsom MD, Kim MO, Schambach A, Schuesler T, Williams DA. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Mol Ther. 2008 Jun; 16(6):1154-1160. PMID: 28178472.
    Citations:    Fields:    
  67. Huang J, Liu Y, Au BC, Barber DL, Arruda A, Schambach A, Rothe M, Minden MD, Paige CJ, Medin JA. Preclinical validation: LV/IL-12 transduction of patient leukemia cells for immunotherapy of AML. Mol Ther Methods Clin Dev. 2016; 3:16074. PMID: 27933304.
    Citations: 2     
  68. Metcalf JA, Ma X, Linders B, Wu S, Schambach A, Ohlemiller KK, Kovacs A, Bigg M, He L, Tollefsen DM, Ponder KP. A Self-inactivating ?-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice. Mol Ther. 2010 Feb; 18(2):334-342. PMID: 28182939.
    Citations:    
  69. 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. Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase. Sci Rep. 2016 12 02; 6:38198. PMID: 27910942.
    Citations: 4     Fields:    Translation:HumansCells
  70. Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ. Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency. Hum Gene Ther. 2017 01; 28(1):112-124. PMID: 27611239.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  71. 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. Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency. Stem Cell Reports. 2016 08 09; 7(2):292-305. PMID: 27453007.
    Citations: 6     Fields:    Translation:AnimalsCells
  72. Schott JW, Morgan M, Galla M, Schambach A. Viral and Synthetic RNA Vector Technologies and Applications. Mol Ther. 2016 09; 24(9):1513-27. PMID: 27377044.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  73. 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. Generation of HLA-Universal iPSC-Derived Megakaryocytes and Platelets for Survival Under Refractoriness Conditions. Mol Med. 2016 Sep; 22:274-285. PMID: 27262025.
    Citations: 6     Fields:    
  74. 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. Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform. Mol Ther. 2016 08; 24(7):1216-26. PMID: 27138041.
    Citations: 1     Fields:    Translation:HumansCells
  75. Garcia-Gomez M, Calabria A, Garcia-Bravo M, Benedicenti F, Kosinski P, López-Manzaneda S, Hill C, Del Mar Mañu-Pereira M, Martín MA, Orman I, Vives-Corrons JL, Kung C, Schambach A, Jin S, Bueren JA, Montini E, Navarro S, Segovia JC. Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency. Mol Ther. 2016 08; 24(7):1187-98. PMID: 27138040.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  76. Labenski V, Suerth JD, Barczak E, Heckl D, Levy C, Bernadin O, Charpentier E, Williams DA, Fehse B, Verhoeyen E, Schambach A. Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes. Biomaterials. 2016 08; 97:97-109. PMID: 27162078.
    Citations: 2     Fields:    Translation:HumansCells
  77. Salari A, Thomay K, Himmler K, Vajen B, Schienke A, Hagedorn M, Ebersold J, Kreipe HH, Krüger A, Schambach A, Schlegelberger B, Göhring G. Establishing a murine xenograft-model for long-term analysis of factors inducing chromosomal instability in myelodysplastic syndrome: Pitfalls and successes. Cancer Genet. 2016 06; 209(6):258-66. PMID: 27184732.
    Citations: 1     Fields:    Translation:HumansAnimals
  78. Manukjan G, Ripperger T, Venturini L, Stadler M, Göhring G, Schambach A, Schlegelberger B, Steinemann D. GABP is necessary for stem/progenitor cell maintenance and myeloid differentiation in human hematopoiesis and chronic myeloid leukemia. Stem Cell Res. 2016 05; 16(3):677-81. PMID: 27100840.
    Citations:    Fields:    Translation:HumansCells
  79. Selich A, Daudert J, Hass R, Philipp F, von Kaisenberg C, Paul G, Cornils K, Fehse B, Rittinghausen S, Schambach A, Rothe M. Massive Clonal Selection and Transiently Contributing Clones During Expansion of Mesenchymal Stem Cell Cultures Revealed by Lentiviral RGB-Barcode Technology. Stem Cells Transl Med. 2016 May; 5(5):591-601. PMID: 27034413.
    Citations: 5     Fields:    Translation:HumansCells
  80. 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. Direct Reprogramming of Hepatic Myofibroblasts into Hepatocytes In Vivo Attenuates Liver Fibrosis. Cell Stem Cell. 2016 06 02; 18(6):797-808. PMID: 26923201.
    Citations: 27     Fields:    Translation:AnimalsCells
  81. Karpinski J, Hauber I, Chemnitz J, Schäfer C, Paszkowski-Rogacz M, Chakraborty D, Beschorner N, Hofmann-Sieber H, Lange UC, Grundhoff A, Hackmann K, Schrock E, Abi-Ghanem J, Pisabarro MT, Surendranath V, Schambach A, Lindner C, van Lunzen J, Hauber J, Buchholz F. Directed evolution of a recombinase that excises the provirus of most HIV-1 primary isolates with high specificity. Nat Biotechnol. 2016 Apr; 34(4):401-9. PMID: 26900663.
    Citations: 17     Fields:    Translation:HumansAnimalsCells
  82. 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. Long noncoding RNA Chast promotes cardiac remodeling. Sci Transl Med. 2016 Feb 17; 8(326):326ra22. PMID: 26888430.
    Citations: 35     Fields:    Translation:HumansAnimalsCells
  83. Papapetrou EP, Schambach A. Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy. Mol Ther. 2016 Apr; 24(4):678-84. PMID: 26867951.
    Citations: 10     Fields:    Translation:Humans
  84. Abbey M, Hakim C, Anand R, Lafera J, Schambach A, Kispert A, Taft MH, Kaever V, Kotlyarov A, Gaestel M, Menon MB. GTPase domain driven dimerization of SEPT7 is dispensable for the critical role of septins in fibroblast cytokinesis. Sci Rep. 2016 Jan 28; 6:20007. PMID: 26818767.
    Citations: 4     Fields:    Translation:AnimalsCells
  85. Thiesler CT, Cajic S, Hoffmann D, Thiel C, van Diepen L, Hennig R, Sgodda M, Weißmann R, Reichl U, Steinemann D, Diekmann U, Huber NM, Oberbeck A, Cantz T, Kuss AW, Körner C, Schambach A, Rapp E, Buettner FF. Glycomic Characterization of Induced Pluripotent Stem Cells Derived from a Patient Suffering from Phosphomannomutase 2 Congenital Disorder of Glycosylation (PMM2-CDG). Mol Cell Proteomics. 2016 Apr; 15(4):1435-52. PMID: 26785728.
    Citations: 6     Fields:    Translation:HumansCells
  86. Maetzig T, Schambach A. Development of Inducible Molecular Switches Based on All-in-One Lentiviral Vectors Equipped with Drug Controlled FLP Recombinase. Methods Mol Biol. 2016; 1448:23-39. PMID: 27317170.
    Citations:    Fields:    Translation:HumansAnimalsCells
  87. Stahlhut M, Schambach A, Kustikova OS. Transduction of Murine Hematopoietic Stem Cells with Tetracycline-regulated Lentiviral Vectors. Methods Mol Biol. 2016; 1448:65-76. PMID: 27317173.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  88. Stahlhut M, Ha TC, Morgan M, Schambach A, Kustikova OS. Comparison of Tetracycline-regulated Promoters in Lentiviral-based Vectors in Murine Transplantation Studies. Curr Gene Ther. 2016; 16(4):242-248. PMID: 27739375.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  89. Schambach A, Morgan M. Retroviral Vectors for Cancer Gene Therapy. Recent Results Cancer Res. 2016; 209:17-35. PMID: 28101685.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  90. Brennig S, Lachmann N, Buchegger T, Hetzel M, Schambach A, Moritz T. Chemoprotection of murine hematopoietic cells by combined gene transfer of cytidine deaminase (CDD) and multidrug resistance 1 gene (MDR1). J Exp Clin Cancer Res. 2015 Dec 12; 34:148. PMID: 26651614.
    Citations: 3     Fields:    Translation:HumansAnimalsCells
  91. Regelin M, Blume J, Pommerencke J, Vakilzadeh R, Witzlau K, Lyszkiewicz M, Zietara N, Saran N, Schambach A, Krueger A. Responsiveness of Developing T Cells to IL-7 Signals Is Sustained by miR-17~92. J Immunol. 2015 Nov 15; 195(10):4832-40. PMID: 26475928.
    Citations: 3     Fields:    Translation:AnimalsCells
  92. Hoffmann D, Göhring G, Heuser M, Ganser A, Schambach A, Morgan MA. Letter to the Editor: Production of Mature Healthy Hematopoietic Cells from Induced Pluripotent Stem Cells Derived from an AML Diagnostic Sample Containing the t(8;21) Translocation. Stem Cells. 2016 Mar; 34(3):797-9. PMID: 26388310.
    Citations:    Fields:    Translation:HumansCells
  93. Schott JW, Hoffmann D, Schambach A. Retrovirus-based vectors for transient and permanent cell modification. Curr Opin Pharmacol. 2015 Oct; 24:135-46. PMID: 26433198.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  94. Suerth JD, Morgan MA, Kloess S, Heckl D, Neudörfl C, Falk CS, Koehl U, Schambach A. Efficient generation of gene-modified human natural killer cells via alpharetroviral vectors. J Mol Med (Berl). 2016 Jan; 94(1):83-93. PMID: 26300042.
    Citations: 8     Fields:    Translation:HumansCells
  95. Dreyer AK, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, Siler U, Reichenbach J, Grez M, Moritz T, Schambach A, Cathomen T. TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells. Biomaterials. 2015 Nov; 69:191-200. PMID: 26295532.
    Citations: 12     Fields:    Translation:HumansCells
  96. Ripperger T, Manukjan G, Meyer J, Wolter S, Schambach A, Bohne J, Modlich U, Li Z, Skawran B, Schlegelberger B, Steinemann D. The heteromeric transcription factor GABP activates the ITGAM/CD11b promoter and induces myeloid differentiation. Biochim Biophys Acta. 2015 Sep; 1849(9):1145-54. PMID: 26170143.
    Citations: 3     Fields:    Translation:HumansAnimalsCells
  97. Heinz N, Ehrnström B, Schambach A, Schwarzer A, Modlich U, Schiedlmeier B. Comparison of Different Cytokine Conditions Reveals Resveratrol as a New Molecule for Ex Vivo Cultivation of Cord Blood-Derived Hematopoietic Stem Cells. Stem Cells Transl Med. 2015 Sep; 4(9):1064-72. PMID: 26160960.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  98. Rissone A, Weinacht KG, la Marca G, Bishop K, Giocaliere E, Jagadeesh J, Felgentreff K, Dobbs K, Al-Herz W, Jones M, Chandrasekharappa S, Kirby M, Wincovitch S, Simon KL, Itan Y, DeVine A, Schlaeger T, Schambach A, Sood R, Notarangelo LD, Candotti F. Reticular dysgenesis-associated AK2 protects hematopoietic stem and progenitor cell development from oxidative stress. J Exp Med. 2015 Jul 27; 212(8):1185-202. PMID: 26150473.
    Citations: 8     Fields:    Translation:AnimalsCells
  99. Dobbs K, Domínguez Conde C, Zhang SY, Parolini S, Audry M, Chou J, Haapaniemi E, Keles S, Bilic I, Okada S, Massaad MJ, Rounioja S, Alwahadneh AM, Serwas NK, Capuder K, Çiftçi E, Felgentreff K, Ohsumi TK, Pedergnana V, Boisson B, Haskologlu S, Ensari A, Schuster M, Moretta A, Itan Y, Patrizi O, Rozenberg F, Lebon P, Saarela J, Knip M, Petrovski S, Goldstein DB, Parrott RE, Savas B, Schambach A, Tabellini G, Bock C, Chatila TA, Comeau AM, Geha RS, Abel L, Buckley RH, Ikinciogullari A, Al-Herz W, Helminen M, Dogu F, Casanova JL, Boztug K, Notarangelo LD. Inherited DOCK2 Deficiency in Patients with Early-Onset Invasive Infections. N Engl J Med. 2015 Jun 18; 372(25):2409-22. PMID: 26083206.
    Citations: 26     Fields:    Translation:HumansCells
  100. Uhlig KM, Schülke S, Scheuplein VA, Malczyk AH, Reusch J, Kugelmann S, Muth A, Koch V, Hutzler S, Bodmer BS, Schambach A, Buchholz CJ, Waibler Z, Scheurer S, Mühlebach MD. Lentiviral Protein Transfer Vectors Are an Efficient Vaccine Platform and Induce a Strong Antigen-Specific Cytotoxic T Cell Response. J Virol. 2015 Sep; 89(17):9044-60. PMID: 26085166.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  101. Stahlhut M, Schwarzer A, Eder M, Yang M, Li Z, Morgan M, Schambach A, Kustikova OS. Lentiviral vector system for coordinated constitutive and drug controlled tetracycline-regulated gene co-expression. Biomaterials. 2015 Sep; 63:189-201. PMID: 26113075.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  102. Manukjan G, Ripperger T, Santer L, von Neuhoff N, Ganser A, Schambach A, Schlegelberger B, Steinemann D. Expression of the ETS transcription factor GABPa is positively correlated to the BCR-ABL1/ABL1 ratio in CML patients and affects imatinib sensitivity in vitro. Exp Hematol. 2015 Oct; 43(10):880-90. PMID: 26072332.
    Citations: 1     Fields:    Translation:HumansCells
  103. Rahman SH, Kuehle J, Reimann C, Mlambo T, Alzubi J, Maeder ML, Riedel H, Fisch P, Cantz T, Rudolph C, Mussolino C, Joung JK, Schambach A, Cathomen T. Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model. PLoS Genet. 2015 May; 11(5):e1005239. PMID: 26000857.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  104. Zhang H, Goudeva L, Immenschuh S, Schambach A, Skokowa J, Eiz-Vesper B, Blasczyk R, Figueiredo C. miR-155 is associated with the leukemogenic potential of the class IV granulocyte colony-stimulating factor receptor in CD34? progenitor cells. Mol Med. 2015 Apr 09; 20:736-46. PMID: 25730818.
    Citations: 4     Fields:    Translation:HumansCells
  105. Coci EG, Maetzig T, Zychlinski D, Rothe M, Suerth JD, Klein C, Schambach A. RETRACTED: Novel and safer self-inactivating vectors for gene therapy of Wiskott-Aldrich Syndrome Curr Gene Ther. 2015 04 09; 15(3):245 - 254. PMID: 25963902.
    Citations:    
  106. Schott JW, Jaeschke NM, Hoffmann D, Maetzig T, Ballmaier M, Godinho T, Cathomen T, Schambach A. Deciphering the impact of parameters influencing transgene expression kinetics after repeated cell transduction with integration-deficient retroviral vectors. Cytometry A. 2015 May; 87(5):405-18. PMID: 25728583.
    Citations: 1     Fields:    Translation:HumansCells
  107. Glienke W, Esser R, Priesner C, Suerth JD, Schambach A, Wels WS, Grez M, Kloess S, Arseniev L, Koehl U. Advantages and applications of CAR-expressing natural killer cells. Front Pharmacol. 2015; 6:21. PMID: 25729364.
    Citations: 51     
  108. 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. Large-scale hematopoietic differentiation of human induced pluripotent stem cells provides granulocytes or macrophages for cell replacement therapies. Stem Cell Reports. 2015 Feb 10; 4(2):282-96. PMID: 25680479.
    Citations: 20     Fields:    Translation:HumansCells
  109. Dahl M, Doyle A, Olsson K, Månsson JE, Marques ARA, Mirzaian M, Aerts JM, Ehinger M, Rothe M, Modlich U, Schambach A, Karlsson S. Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice. Mol Ther. 2015 May; 23(5):835-844. PMID: 25655314.
    Citations: 8     Fields:    Translation:HumansAnimalsCells
  110. Müller-Kuller U, Ackermann M, Kolodziej S, Brendel C, Fritsch J, Lachmann N, Kunkel H, Lausen J, Schambach A, Moritz T, Grez M. A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells. Nucleic Acids Res. 2015 Feb 18; 43(3):1577-92. PMID: 25605798.
    Citations: 14     Fields:    Translation:HumansAnimalsCells
  111. Retraction Notice Curr Gene Ther. 2015; 15(3):245 - 254. PMID: 25619887.
    Citations: 3     Fields:    
  112. Carmo M, Risma KA, Arumugam P, Tiwari S, Hontz AE, Montiel-Equihua CA, Alonso-Ferrero ME, Blundell MP, Schambach A, Baum C, Malik P, Thrasher AJ, Jordan MB, Gaspar HB. Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency. Mol Ther. 2015 Apr; 23(4):737-45. PMID: 25523759.
    Citations: 6     Fields:    Translation:AnimalsCells
  113. Suerth JD, Labenski V, Schambach A. Alpharetroviral vectors: from a cancer-causing agent to a useful tool for human gene therapy. Viruses. 2014 Dec 05; 6(12):4811-38. PMID: 25490763.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  114. Lyszkiewicz M, Zietara N, Föhse L, Puchalka J, Diestelhorst J, Witzlau K, Prinz I, Schambach A, Krueger A. Limited niche availability suppresses murine intrathymic dendritic-cell development from noncommitted progenitors. Blood. 2015 Jan 15; 125(3):457-64. PMID: 25411428.
    Citations: 5     Fields:    Translation:AnimalsCells
  115. Coci EG, Thau-Habermann N, Maetzig T, Li Z, Klein C, Petri S, Schambach A. TEMPORARY REMOVAL: Assessment of hematopoietic and neurologic pathophysiology of HCLS1-associated protein X-1 deficiency in a Hax1-knockout mouse model. Exp Hematol. 2014 Oct 28. PMID: 25448489.
    Citations:    Fields:    
  116. Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. PMID: 25295500.
    Citations: 65     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
  117. Liu L, Xu Y, He M, Zhang M, Cui F, Lu L, Yao M, Tian W, Benda C, Zhuang Q, Huang Z, Li W, Li X, Zhao P, Fan W, Luo Z, Li Y, Wu Y, Hutchins AP, Wang D, Tse HF, Schambach A, Frampton J, Qin B, Bao X, Yao H, Zhang B, Sun H, Pei D, Wang H, Wang J, Esteban MA. Transcriptional pause release is a rate-limiting step for somatic cell reprogramming. Cell Stem Cell. 2014 Nov 06; 15(5):574-88. PMID: 25312495.
    Citations: 20     Fields:    Translation:HumansAnimalsCells
  118. Haemmerle R, Phaltane R, Rothe M, Schröder S, Schambach A, Moritz T, Modlich U. Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model. Mol Ther Nucleic Acids. 2014 Oct 07; 3:e200. PMID: 25291142.
    Citations: 5     
  119. Jaako P, Debnath S, Olsson K, Modlich U, Rothe M, Schambach A, Flygare J, Karlsson S. Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia. Haematologica. 2014 Dec; 99(12):1792-8. PMID: 25216681.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  120. Wang J, Sun Q, Morita Y, Jiang H, Groß A, Lechel A, Hildner K, Guachalla LM, Gompf A, Hartmann D, Schambach A, Wüstefeld T, Dauch D, Schrezenmeier H, Hofmann WK, Nakauchi H, Ju Z, Kestler HA, Zender L, Rudolph KL. A Differentiation Checkpoint Limits Hematopoietic Stem Cell Self-Renewal in Response to DNA Damage. Cell. 2014 Sep 11; 158(6):1444. PMID: 28915370.
    Citations: 1     Fields:    
  121. Menon MB, Sawada A, Chaturvedi A, Mishra P, Schuster-Gossler K, Galla M, Schambach A, Gossler A, Förster R, Heuser M, Kotlyarov A, Kinoshita M, Gaestel M. Genetic deletion of SEPT7 reveals a cell type-specific role of septins in microtubule destabilization for the completion of cytokinesis. PLoS Genet. 2014 Aug; 10(8):e1004558. PMID: 25122120.
    Citations: 22     Fields:    Translation:HumansAnimalsCells
  122. Schambach A. Toward a safer integration profile of MLV-based retroviral vectors. Mol Ther. 2014 Aug; 22(8):1405-1406. PMID: 25082089.
    Citations: 1     Fields:    
  123. Warlich E, Schambach A, Lock D, Wedekind D, Glage S, Eckardt D, Bosio A, Knöbel S. FAS-based cell depletion facilitates the selective isolation of mouse induced pluripotent stem cells. PLoS One. 2014; 9(7):e102171. PMID: 25029550.
    Citations:    Fields:    Translation:HumansAnimalsCells
  124. Huang J, Guo X, Fan N, Song J, Zhao B, Ouyang Z, Liu Z, Zhao Y, Yan Q, Yi X, Schambach A, Frampton J, Esteban MA, Yang D, Yang H, Lai L. RAG1/2 knockout pigs with severe combined immunodeficiency. J Immunol. 2014 Aug 01; 193(3):1496-503. PMID: 24973446.
    Citations: 26     Fields:    Translation:AnimalsCells
  125. Pike-Overzet K, Baum C, Bredius RG, Cavazzana M, Driessen GJ, Fibbe WE, Gaspar HB, Hoeben RC, Lagresle-Peyrou C, Lankester A, Meij P, Schambach A, Thrasher A, Van Dongen JJ, Zwaginga JJ, Staal FJ. Successful RAG1-SCID gene therapy depends on the level of RAG1 expression. J Allergy Clin Immunol. 2014 Jul; 134(1):242-3. PMID: 25117803.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  126. Limbourg A, Schnabel S, Lozanovski VJ, Napp LC, Ha TC, Maetzig T, Bauersachs J, Naim HY, Schambach A, Limbourg FP. Genetic reporter analysis reveals an expandable reservoir of OCT4+ cells in adult skin. Cell Regen (Lond). 2014; 3(1):9. PMID: 25408888.
    Citations:    
  127. Turan S, Qiao J, Madden S, Benham C, Kotz M, Schambach A, Bode J. Expanding Flp-RMCE options: the potential of Recombinase Mediated Twin-Site Targeting (RMTT). Gene. 2014 Aug 10; 546(2):135-44. PMID: 24905650.
    Citations: 3     Fields:    Translation:AnimalsCells
  128. 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. Differential role of nonhomologous end joining factors in the generation, DNA damage response, and myeloid differentiation of human induced pluripotent stem cells. Proc Natl Acad Sci U S A. 2014 Jun 17; 111(24):8889-94. PMID: 24889605.
    Citations: 12     Fields:    Translation:HumansCells
  129. Moiani A, Suerth JD, Gandolfi F, Rizzi E, Severgnini M, De Bellis G, Schambach A, Mavilio F. Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells. Genes (Basel). 2014 May 16; 5(2):415-29. PMID: 24840152.
    Citations: 5     
  130. Emmrich S, Rasche M, Schöning J, Reimer C, Keihani S, Maroz A, Xie Y, Li Z, Schambach A, Reinhardt D, Klusmann JH. miR-99a/100~125b tricistrons regulate hematopoietic stem and progenitor cell homeostasis by shifting the balance between TGFß and Wnt signaling. Genes Dev. 2014 Apr 15; 28(8):858-74. PMID: 24736844.
    Citations: 40     Fields:    Translation:HumansAnimalsCells
  131. Bobis-Wozowicz S, Galla M, Alzubi J, Kuehle J, Baum C, Schambach A, Cathomen T. Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery. Sci Rep. 2014 Apr 11; 4:4656. PMID: 24722320.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  132. Kustikova OS, Stahlhut M, Ha TC, Scherer R, Schambach A, Baum C. Dose response and clonal variability of lentiviral tetracycline-regulated vectors in murine hematopoietic cells. Exp Hematol. 2014 Jul; 42(7):505-515.e7. PMID: 24674753.
    Citations: 3     Fields:    Translation:AnimalsCells
  133. 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. Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci Transl Med. 2014 Mar 12; 6(227):227ra33. PMID: 24622513.
    Citations: 100     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
  134. Maetzig T, Kuehle J, Schwarzer A, Turan S, Rothe M, Chaturvedi A, Morgan M, Ha TC, Heuser M, Hammerschmidt W, Baum C, Schambach A. All-in-One inducible lentiviral vector systems based on drug controlled FLP recombinase. Biomaterials. 2014 May; 35(14):4345-56. PMID: 24529624.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  135. Wunderlich S, Kircher M, Vieth B, Haase A, Merkert S, Beier J, Göhring G, Glage S, Schambach A, Curnow EC, Pääbo S, Martin U, Enard W. Primate iPS cells as tools for evolutionary analyses. Stem Cell Res. 2014 May; 12(3):622-9. PMID: 24631741.
    Citations: 13     Fields:    Translation:HumansAnimalsCells
  136. Kuehle J, Turan S, Cantz T, Hoffmann D, Suerth JD, Maetzig T, Zychlinski D, Klein C, Steinemann D, Baum C, Bode J, Schambach A. Modified lentiviral LTRs allow Flp recombinase-mediated cassette exchange and in vivo tracing of "factor-free" induced pluripotent stem cells. Mol Ther. 2014 May; 22(5):919-28. PMID: 24434935.
    Citations: 13     Fields:    Translation:AnimalsCells
  137. Lachmann N, Happle C, Ackermann M, Lüttge D, Wetzke M, Merkert S, Hetzel M, Kensah G, Jara-Avaca M, Mucci A, Skuljec J, Dittrich AM, Pfaff N, Brennig S, Schambach A, Steinemann D, Göhring G, Cantz T, Martin U, Schwerk N, Hansen G, Moritz T. Gene correction of human induced pluripotent stem cells repairs the cellular phenotype in pulmonary alveolar proteinosis. Am J Respir Crit Care Med. 2014 Jan 15; 189(2):167-82. PMID: 24279725.
    Citations: 22     Fields:    Translation:HumansCells
  138. Rothe M, Schambach A, Biasco L. Safety of gene therapy: new insights to a puzzling case. Curr Gene Ther. 2014; 14(6):429-36. PMID: 25245088.
    Citations: 9     Fields:    Translation:HumansAnimals
  139. Rothe M, Modlich U, Schambach A. Biosafety challenges for use of lentiviral vectors in gene therapy. Curr Gene Ther. 2013 Dec; 13(6):453-68. PMID: 24195603.
    Citations: 16     Fields:    Translation:HumansAnimalsCells
  140. 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. Promoter and lineage independent anti-silencing activity of the A2 ubiquitous chromatin opening element for optimized human pluripotent stem cell-based gene therapy. Biomaterials. 2014 Feb; 35(5):1531-42. PMID: 24290698.
    Citations: 15     Fields:    Translation:HumansAnimalsCells
  141. Zwingenberger S, Yao Z, Jacobi A, Vater C, Valladares RD, Li C, Nich C, Rao AJ, Christman JE, Antonios JK, Gibon E, Schambach A, Maetzig T, Goodman SB, Stiehler M. Enhancement of BMP-2 induced bone regeneration by SDF-1a mediated stem cell recruitment. Tissue Eng Part A. 2014 Feb; 20(3-4):810-8. PMID: 24090366.
    Citations: 11     Fields:    Translation:HumansAnimalsCells
  142. Hauber I, Hofmann-Sieber H, Chemnitz J, Dubrau D, Chusainow J, Stucka R, Hartjen P, Schambach A, Ziegler P, Hackmann K, Schröck E, Schumacher U, Lindner C, Grundhoff A, Baum C, Manz MG, Buchholz F, Hauber J. Highly significant antiviral activity of HIV-1 LTR-specific tre-recombinase in humanized mice. PLoS Pathog. 2013; 9(9):e1003587. PMID: 24086129.
    Citations: 20     Fields:    Translation:HumansAnimalsCells
  143. Gupta SS, Maetzig T, Maertens GN, Sharif A, Rothe M, Weidner-Glunde M, Galla M, Schambach A, Cherepanov P, Schulz TF. Bromo- and extraterminal domain chromatin regulators serve as cofactors for murine leukemia virus integration. J Virol. 2013 Dec; 87(23):12721-36. PMID: 24049186.
    Citations: 57     Fields:    Translation:HumansAnimalsCells
  144. Kaufmann KB, Büning H, Galy A, Schambach A, Grez M. Gene therapy on the move. EMBO Mol Med. 2013 11; 5(11):1642-61. PMID: 24106209.
    Citations: 34     Fields:    Translation:Humans
  145. Mandel K, Yang Y, Schambach A, Glage S, Otte A, Hass R. Mesenchymal stem cells directly interact with breast cancer cells and promote tumor cell growth in vitro and in vivo. Stem Cells Dev. 2013 Dec 01; 22(23):3114-27. PMID: 23895436.
    Citations: 27     Fields:    Translation:HumansAnimalsCells
  146. Eggenschwiler R, Loya K, Wu G, Sharma AD, Sgodda M, Zychlinski D, Herr C, Steinemann D, Teckman J, Bals R, Ott M, Schambach A, Schöler HR, Cantz T. Sustained knockdown of a disease-causing gene in patient-specific induced pluripotent stem cells using lentiviral vector-based gene therapy. Stem Cells Transl Med. 2013 Sep; 2(9):641-54. PMID: 23926210.
    Citations: 11     Fields:    Translation:HumansAnimalsCells
  147. Schambach A, Moritz T. Toward position-independent retroviral vector expression in pluripotent stem cells. Mol Ther. 2013 Aug; 21(8):1474-7. PMID: 23903574.
    Citations:    Fields:    Translation:AnimalsCells
  148. Gürlevik E, Fleischmann-Mundt B, Armbrecht N, Longerich T, Woller N, Kloos A, Hoffmann D, Schambach A, Wirth TC, Manns MP, Zender L, Kubicka S, Kühnel F. Adjuvant gemcitabine therapy improves survival in a locally induced, R0-resectable model of metastatic intrahepatic cholangiocarcinoma. Hepatology. 2013 Sep; 58(3):1031-41. PMID: 23686746.
    Citations: 9     Fields:    Translation:Animals
  149. 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, Sandusky GE, Kunkel H, Rudolf E, Dillmann A, von Kalle C, Kühlcke K, Baum C, Schambach A, Dinauer MC, Schmidt M, Grez M. From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease. Hum Gene Ther Clin Dev. 2013 Jun; 24(2):86-98. PMID: 23845071.
    Citations: 9     Fields:    Translation:HumansAnimalsCells
  150. Rittelmeyer I, Rothe M, Brugman MH, Iken M, Schambach A, Manns MP, Baum C, Modlich U, Ott M. Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents. Hepatology. 2013 Jul; 58(1):397-408. PMID: 23258554.
    Citations: 12     Fields:    Translation:AnimalsCells
  151. Brugman MH, Suerth JD, Rothe M, Suerbaum S, Schambach A, Modlich U, Kustikova O, Baum C. Evaluating a ligation-mediated PCR and pyrosequencing method for the detection of clonal contribution in polyclonal retrovirally transduced samples. Hum Gene Ther Methods. 2013 Apr; 24(2):68-79. PMID: 23384086.
    Citations: 12     Fields:    Translation:AnimalsCells
  152. Pfaff N, Lachmann N, Ackermann M, Kohlscheen S, Brendel C, Maetzig T, Niemann H, Antoniou MN, Grez M, Schambach A, Cantz T, Moritz T. A ubiquitous chromatin opening element prevents transgene silencing in pluripotent stem cells and their differentiated progeny. Stem Cells. 2013 Mar; 31(3):488-99. PMID: 23307570.
    Citations: 27     Fields:    Translation:HumansAnimalsCells
  153. Kotlarz D, Zietara N, Uzel G, Weidemann T, Braun CJ, Diestelhorst J, Krawitz PM, Robinson PN, Hecht J, Puchalka J, Gertz EM, Schäffer AA, Lawrence MG, Kardava L, Pfeifer D, Baumann U, Pfister ED, Hanson EP, Schambach A, Jacobs R, Kreipe H, Moir S, Milner JD, Schwille P, Mundlos S, Klein C. Loss-of-function mutations in the IL-21 receptor gene cause a primary immunodeficiency syndrome. J Exp Med. 2013 Mar 11; 210(3):433-43. PMID: 23440042.
    Citations: 51     Fields:    Translation:HumansCells
  154. Sundarasetty BS, Singh VK, Salguero G, Geffers R, Rickmann M, Macke L, Borchers S, Figueiredo C, Schambach A, Gullberg U, Provasi E, Bonini C, Ganser A, Woelfel T, Stripecke R. Lentivirus-induced dendritic cells for immunization against high-risk WT1(+) acute myeloid leukemia. Hum Gene Ther. 2013 Feb; 24(2):220-37. PMID: 23311414.
    Citations: 6     Fields:    Translation:HumansAnimalsCells
  155. Schambach A, Zychlinski D, Ehrnstroem B, Baum C. Biosafety features of lentiviral vectors. Hum Gene Ther. 2013 Feb; 24(2):132-42. PMID: 23311447.
    Citations: 21     Fields:    Translation:HumansCells
  156. Galla M, Schambach A, Baum C. Retrovirus-based mRNA transfer for transient cell manipulation. Methods Mol Biol. 2013; 969:139-61. PMID: 23296933.
    Citations: 4     Fields:    Translation:AnimalsCells
  157. Grabundzija I, Wang J, Sebe A, Erdei Z, Kajdi R, Devaraj A, Steinemann D, Szuhai K, Stein U, Cantz T, Schambach A, Baum C, Izsvák Z, Sarkadi B, Ivics Z. Sleeping Beauty transposon-based system for cellular reprogramming and targeted gene insertion in induced pluripotent stem cells. Nucleic Acids Res. 2013 Feb 01; 41(3):1829-47. PMID: 23275558.
    Citations: 22     Fields:    Translation:HumansAnimalsCells
  158. Zwingenberger S, Yao Z, Jacobi A, Vater C, Valladares RD, Li C, Nich C, Rao AJ, Christman JE, Antonios JK, Gibon E, Schambach A, Mätzig T, Günther KP, Goodman SB, Stiehler M. Stem cell attraction via SDF-1a expressing fat tissue grafts. J Biomed Mater Res A. 2013 Jul; 101(7):2067-74. PMID: 23281045.
    Citations: 3     Fields:    Translation:AnimalsCells
  159. Kaufmann KB, Brendel C, Suerth JD, Mueller-Kuller U, Chen-Wichmann L, Schwäble J, Pahujani S, Kunkel H, Schambach A, Baum C, Grez M. Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing. Mol Ther. 2013 Mar; 21(3):648-61. PMID: 23207695.
    Citations: 11     Fields:    Translation:HumansAnimalsCells
  160. Wiedemann A, Hemmer K, Bernemann I, Göhring G, Pogozhykh O, Figueiredo C, Glage S, Schambach A, Schwamborn JC, Blasczyk R, Müller T. Induced pluripotent stem cells generated from adult bone marrow-derived cells of the nonhuman primate (Callithrix jacchus) using a novel quad-cistronic and excisable lentiviral vector. Cell Reprogram. 2012 Dec; 14(6):485-96. PMID: 23194452.
    Citations: 10     Fields:    Translation:AnimalsCells
  161. Wunderlich S, Haase A, Merkert S, Beier J, Schwanke K, Schambach A, Glage S, Göhring G, Curnow EC, Martin U. Induction of pluripotent stem cells from a cynomolgus monkey using a polycistronic simian immunodeficiency virus-based vector, differentiation toward functional cardiomyocytes, and generation of stably expressing reporter lines. Cell Reprogram. 2012 Dec; 14(6):471-84. PMID: 23194451.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  162. Maetzig T, Baum C, Schambach A. Retroviral protein transfer: falling apart to make an impact. Curr Gene Ther. 2012 Oct; 12(5):389-409. PMID: 22934617.
    Citations: 4     Fields:    Translation:HumansAnimalsCells