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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. Delville M, Bellier F, Leon J, Klifa R, Lizot S, Vinçon H, Sobrino S, Thouenon R, Marchal A, Garrigue A, Olivré J, Charbonnier S, Lagresle-Peyrou C, Amendola M, Schambach A, Gross D, Lamarthée B, Benoist C, Zuber J, André I, Cavazzana M, Six E. A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome. Blood. 2021 Apr 29; 137(17):2326-2336. PMID: 33545713.
    Citations:    Fields:    
  2. Kosanke M, Osetek K, Haase A, Wiehlmann L, Davenport C, Schwarzer A, Adams F, Kleppa MJ, Schambach A, Merkert S, Wunderlich S, Menke S, Dorda M, Martin U. Reprogramming enriches for somatic cell clones with small-scale mutations in cancer-associated genes. Mol Ther. 2021 Apr 06. PMID: 33831558.
    Citations:    Fields:    
  3. Morgan MA, Galla M, Grez M, Fehse B, Schambach A. Retroviral gene therapy in Germany with a view on previous experience and future perspectives. Gene Ther. 2021 Mar 22. PMID: 33753908.
    Citations:    Fields:    
  4. Hoffmann D, Sens J, Brennig S, Brand D, Philipp F, Vollmer Barbosa P, Kuehle J, Steinemann D, Lenz D, Buchegger T, Morgan M, Falk CS, Klein C, Lachmann N, Schambach A. Genetic Correction of IL-10RB Deficiency Reconstitutes Anti-Inflammatory Regulation in iPSC-Derived Macrophages. J Pers Med. 2021 Mar 20; 11(3). PMID: 33804706.
    Citations:    
  5. Schejtman A, Vetharoy W, Choi U, Rivat C, Theobald N, Piras G, Leon-Rico D, Buckland K, Armenteros-Monterroso E, Benedetti S, Ashworth MT, Rothe M, Schambach A, Gaspar HB, Kang E, Malech HL, Thrasher AJ, Santilli G. Pre-clinical optimisation and safety studies of a new lentiviral gene therapy for p47phox-deficient chronic granulomatous disease. Hum Gene Ther. 2021 Mar 19. PMID: 33740872.
    Citations:    Fields:    
  6. Stahlhut M, Ha TC, Takmakova E, Morgan MA, Schwarzer A, Schaudien D, Eder M, Schambach A, Kustikova OS. Conditionally immortalised leukaemia initiating cells co-expressing Hoxa9/Meis1 demonstrate microenvironmental adaptation properties ex vivo while maintaining myelomonocytic memory. Sci Rep. 2021 Mar 05; 11(1):5294. PMID: 33674652.
    Citations:    Fields:    
  7. Lange L, Morgan M, Schambach A. The hemogenic endothelium: a critical source for the generation of PSC-derived hematopoietic stem and progenitor cells. Cell Mol Life Sci. 2021 Feb 09. PMID: 33559689.
    Citations:    Fields:    
  8. Liu Y, Dahl M, Debnath S, Rothe M, Smith EM, Grahn THM, Warsi S, Chen J, Flygare J, Schambach A, Karlsson S. Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector. Haematologica. 2021 Jan 14. PMID: 33440921.
    Citations:    Fields:    
  9. Sens J, Hoffmann D, Lange L, Vollmer Barbosa P, Morgan M, Falk CS, Schambach A. Knockout-Induced Pluripotent Stem Cells for Disease and Therapy Modeling of IL-10-Associated Primary Immunodeficiencies. Hum Gene Ther. 2021 Jan; 32(1-2):77-95. PMID: 33023317.
    Citations: 1     Fields:    
  10. Moscatelli I, Almarza E, Schambach A, Ricks D, Schulz A, Herzog CD, Henriksen K, Askmyr M, Schwartz JD, Richter J. Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:389-397. PMID: 33575431.
    Citations:    
  11. Ha TC, Stahlhut M, Rothe M, Paul G, Dziadek V, Morgan M, Brugman M, Fehse B, Kustikova O, Schambach A, Baum C. Multiple Genes Surrounding Bcl-xL, a Common Retroviral Insertion Site, Can Influence Hematopoiesis Individually or in Concert. Hum Gene Ther. 2020 Dec 07. PMID: 33012194.
    Citations: 1     Fields:    
  12. Dahl M, Smith EMK, Warsi S, Rothe M, Ferraz MJ, Aerts JMFG, Golipour A, Harper C, Pfeifer R, Pizzurro D, Schambach A, Mason C, Karlsson S. Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:312-323. PMID: 33511245.
    Citations:    
  13. Brinkert K, Hedtfeld S, Burhop A, Gastmeier R, Gad P, Wedekind D, Kloth C, Rothschuh J, Lachmann N, Hetzel M, Jirmo AC, Lopez-Rodriguez E, Brandenberger C, Hansen G, Schambach A, Ackermann M, Tümmler B, Munder A. Rescue from Pseudomonas aeruginosa Airway Infection via Stem Cell Transplantation. Mol Ther. 2021 03 03; 29(3):1324-1334. PMID: 33279724.
    Citations: 1     Fields:    
  14. Paredes R, Kelly JR, Geary B, Almarzouq B, Schneider M, Pearson S, Narayanan P, Williamson A, Lovell SC, Wiseman DH, Chadwick JA, Jones NJ, Kustikova O, Schambach A, Garner T, Amaral FMR, Pierce A, Stevens A, Somervaille TCP, Whetton AD, Meyer S. EVI1 phosphorylation at S436 regulates interactions with CtBP1 and DNMT3A and promotes self-renewal. Cell Death Dis. 2020 10 20; 11(10):878. PMID: 33082307.
    Citations:    Fields:    
  15. Dragon AC, Zimmermann K, Nerreter T, Sandfort D, Lahrberg J, Klöß S, Kloth C, Mangare C, Bonifacius A, Tischer-Zimmermann S, Blasczyk R, Maecker-Kolhoff B, Uchanska-Ziegler B, Abken H, Schambach A, Hudecek M, Eiz-Vesper B. CAR-T cells and TRUCKs that recognize an EBNA-3C-derived epitope presented on HLA-B*35 control Epstein-Barr virus-associated lymphoproliferation. J Immunother Cancer. 2020 10; 8(2). PMID: 33127653.
    Citations:    Fields:    
  16. Klatt D, Ha TC, Schinke M, Selich A, Lieske A, Dahlke J, Morgan M, Maetzig T, Schambach A. Competitive sgRNA Screen Identifies p38 MAPK as a Druggable Target to Improve HSPC Engraftment. Cells. 2020 09 29; 9(10). PMID: 33003308.
    Citations: 1     Fields:    
  17. Sgodda M, Alfken S, Schambach A, Eggenschwiler R, Fidzinski P, Hummel M, Cantz T. Synthetic Notch-Receptor-Mediated Transmission of a Transient Signal into Permanent Information via CRISPR/Cas9-Based Genome Editing. Cells. 2020 08 20; 9(9). PMID: 32825374.
    Citations:    Fields:    Translation:Humans
  18. Morgan MA, Büning H, Sauer M, Schambach A. Use of Cell and Genome Modification Technologies to Generate Improved "Off-the-Shelf" CAR T and CAR NK Cells. Front Immunol. 2020; 11:1965. PMID: 32903482.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  19. Ritter M, Klimiankou M, Klimenkova O, Schambach A, Hoffmann D, Schmidt A, Kanz L, Link DC, Welte K, Skokowa J. Cooperating, congenital neutropenia-associated Csf3r and Runx1 mutations activate pro-inflammatory signaling and inhibit myeloid differentiation of mouse HSPCs. Ann Hematol. 2020 Oct; 99(10):2329-2338. PMID: 32821971.
    Citations: 1     Fields:    Translation:AnimalsCells
  20. 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 07 14; 15(1):274. PMID: 32668220.
    Citations:    Fields:    
  21. Wolfien M, Klatt D, Salybekov AA, Ii M, Komatsu-Horii M, Gaebel R, Philippou-Massier J, Schrinner E, Akimaru H, Akimaru E, David R, Garbade J, Gummert J, Haverich A, Hennig H, Iwasaki H, Kaminski A, Kawamoto A, Klopsch C, Kowallick JT, Krebs S, Nesteruk J, Reichenspurner H, Ritter C, Stamm C, Tani-Yokoyama A, Blum H, Wolkenhauer O, Schambach A, Asahara T, Steinhoff G. Hematopoietic stem-cell senescence and myocardial repair - Coronary artery disease genotype/phenotype analysis of post-MI myocardial regeneration response induced by CABG/CD133+ bone marrow hematopoietic stem cell treatment in RCT PERFECT Phase 3. EBioMedicine. 2020 Jul; 57:102862. PMID: 32629392.
    Citations: 3     Fields:    
  22. Kloos A, Mintzas K, Winckler L, Gabdoulline R, Alwie Y, Jyotsana N, Kattre N, Schottmann R, Scherr M, Gupta C, Adams FF, Schwarzer A, Heckl D, Schambach A, Imren S, Humphries RK, Ganser A, Thol F, Heuser M. Effective drug treatment identified by in vivo screening in a transplantable patient-derived xenograft model of chronic myelomonocytic leukemia. Leukemia. 2020 11; 34(11):2951-2963. PMID: 32576961.
    Citations:    Fields:    Translation:HumansAnimals
  23. 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 09; 73:109672. PMID: 32428553.
    Citations: 2     Fields:    
  24. 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: 2     Fields:    Translation:HumansCells
  25. 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     
  26. 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: 3     
  27. 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 06; 27(6):297-306. PMID: 32051561.
    Citations: 1     Fields:    
  28. 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: 10     
  29. 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: 12     Fields:    Translation:HumansCells
  30. 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 02; 31(3-4):183-198. PMID: 31760808.
    Citations: 2     Fields:    
  31. 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 02; 31(3-4):199-210. PMID: 31773990.
    Citations: 2     Fields:    
  32. 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: 4     Fields:    
  33. 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: 7     
  34. 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: 6     Fields:    Translation:HumansAnimalsCells
  35. 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: 4     Fields:    Translation:HumansAnimalsCells
  36. 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: 4     Fields:    Translation:HumansCells
  37. 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: 3     
  38. 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: 6     Fields:    Translation:HumansAnimalsCells
  39. 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 04; 105(4):1147-1157. PMID: 31289207.
    Citations: 1     Fields:    Translation:AnimalsCells
  40. 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: 3     Fields:    Translation:HumansAnimalsCells
  41. 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: 11     
  42. 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: 11     Fields:    Translation:HumansCells
  43. 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: 9     Fields:    Translation:HumansAnimalsCells
  44. 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: 9     Fields:    Translation:HumansCells
  45. 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: 20     Fields:    Translation:HumansAnimalsCells
  46. 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: 3     
  47. Morgan MA, Schambach A. Engineering CAR-T Cells for Improved Function Against Solid Tumors. Front Immunol. 2018; 9:2493. PMID: 30420856.
    Citations: 17     Fields:    Translation:HumansCells
  48. 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: 3     Fields:    Translation:HumansAnimalsCells
  49. 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:    
  50. 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: 8     
  51. 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: 5     Fields:    Translation:HumansAnimalsCells
  52. 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: 4     Fields:    Translation:HumansAnimalsCells
  53. 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: 4     Fields:    Translation:AnimalsCells
  54. Maetzig T, Morgan M, Schambach A. Fluorescent genetic barcoding for cellular multiplex analyses. Exp Hematol. 2018 11; 67:10-17. PMID: 30098396.
    Citations:    Fields:    Translation:HumansAnimalsCells
  55. 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: 2     Fields:    Translation:HumansAnimalsCells
  56. 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: 1     Fields:    
  57. 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: 8     Fields:    Translation:HumansAnimalsCells
  58. 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: 11     
  59. 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: 42     Fields:    Translation:HumansCells
  60. 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: 2     Fields:    Translation:HumansCells
  61. 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: 9     Fields:    Translation:HumansAnimalsCells
  62. 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: 4     Fields:    Translation:HumansCells
  63. 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: 6     Fields:    Translation:AnimalsCells
  64. 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
  65. 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: 2     Fields:    
  66. 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: 12     
  67. 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: 4     Fields:    Translation:HumansAnimalsCells
  68. 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: 9     Fields:    Translation:HumansAnimalsCells
  69. 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: 6     Fields:    Translation:HumansAnimalsCells
  70. 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: 3     Fields:    Translation:HumansAnimalsCells
  71. 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: 14     Fields:    Translation:HumansAnimalsCells
  72. 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: 24     Fields:    Translation:HumansCells
  73. 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: 1     Fields:    Translation:AnimalsCells
  74. 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: 41     Fields:    Translation:HumansCells
  75. 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: 1     Fields:    Translation:AnimalsCells
  76. 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: 4     Fields:    
  77. 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: 3     Fields:    Translation:AnimalsCells
  78. 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: 11     Fields:    Translation:HumansAnimalsCells
  79. 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: 4     Fields:    Translation:HumansCells
  80. 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: 12     
  81. 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: 3     Fields:    Translation:HumansAnimalsCells
  82. 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: 6     Fields:    Translation:HumansCells
  83. 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: 8     Fields:    Translation:HumansAnimalsCells
  84. 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: 4     Fields:    Translation:HumansAnimalsCells
  85. 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: 52     Fields:    Translation:HumansAnimalsCells
  86. 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: 4     Fields:    Translation:AnimalsCells
  87. 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: 5     Fields:    Translation:HumansCells
  88. 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: 1     Fields:    
  89. 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: 7     
  90. 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:    
  91. 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: 9     Fields:    Translation:HumansCells
  92. 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: 12     Fields:    Translation:HumansAnimalsCells
  93. 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: 10     Fields:    Translation:AnimalsCells
  94. 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: 14     Fields:    Translation:HumansAnimalsCells
  95. 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: 24     Fields:    
  96. 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: 7     Fields:    Translation:HumansCells
  97. 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: 11     Fields:    Translation:HumansAnimalsCells
  98. 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: 5     Fields:    Translation:HumansCells
  99. 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: 2     Fields:    Translation:HumansAnimals
  100. 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: 2     Fields:    Translation:HumansCells
  101. 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: 23     Fields:    Translation:HumansCells
  102. 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: 50     Fields:    Translation:AnimalsCells
  103. 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: 34     Fields:    Translation:HumansAnimalsCells
  104. 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: 95     Fields:    Translation:HumansAnimalsCells
  105. 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: 45     Fields:    Translation:Humans
  106. 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: 9     Fields:    Translation:AnimalsCells
  107. 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