Harvard Catalyst Profiles

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Axel Schambach, Ph.D.

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selected publications
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. Paasch D, Meyer J, Stamopoulou A, Lenz D, Kuehle J, Kloos D, Buchegger T, Holzinger A, Falk CS, Kloth C, von Kaisenberg CS, Abken H, Schambach A, Lachmann N, Morgan M, Moritz T. Ex Vivo Generation of CAR Macrophages from Hematopoietic Stem and Progenitor Cells for Use in Cancer Therapy. Cells. 2022 03 15; 11(6). PMID: 35326445.
    Citations:    Fields:    
  2. 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. 2022 02 01; 107(2):446-456. PMID: 33440921.
    Citations: 3     Fields:    
  3. Baron Y, Sens J, Lange L, Nassauer L, Klatt D, Hoffmann D, Kleppa MJ, Barbosa PV, Keisker M, Steinberg V, Suerth JD, Vondran FWR, Meyer J, Morgan M, Schambach A, Galla M. Improved alpharetrovirus-based Gag.MS2 particles for efficient and transient delivery of CRISPR-Cas9 into target cells. Mol Ther Nucleic Acids. 2022 Mar 08; 27:810-823. PMID: 35141043.
    Citations: 1     
  4. Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Betibeglogene Autotemcel Gene Therapy for Non-ß0/ß0 Genotype ß-Thalassemia. N Engl J Med. 2022 02 03; 386(5):415-427. PMID: 34891223.
    Citations:    Fields:    Translation:HumansCellsCTClinical Trials
  5. 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. Correction: Effective drug treatment identified by in vivo screening in a transplantable patient-derived xenograft model of chronic myelomonocytic leukemia. Leukemia. 2021 Dec; 35(12):3629. PMID: 34728793.
    Citations:    Fields:    
  6. Rudek LS, Zimmermann K, Galla M, Meyer J, Kuehle J, Stamopoulou A, Brand D, Sandalcioglu IE, Neyazi B, Moritz T, Rossig C, Altvater B, Falk CS, Abken H, Morgan MA, Schambach A. Generation of an NF?B-Driven Alpharetroviral "All-in-One" Vector Construct as a Potent Tool for CAR NK Cell Therapy. Front Immunol. 2021; 12:751138. PMID: 34804035.
    Citations:    Fields:    Translation:HumansCells
  7. Büning H, Fehse B, Ivics Z, Kochanek S, Koehl U, Kupatt C, Mussolino C, Nettelbeck DM, Schambach A, Uckert W, Wagner E, Cathomen T. Gene Therapy "Made in Germany": A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene Therapy. Hum Gene Ther. 2021 Oct; 32(19-20):987-996. PMID: 34662229.
    Citations:    Fields:    Translation:Humans
  8. Lieske A, Ha TC, Schambach A, Maetzig T. An Improved Lentiviral Fluorescent Genetic Barcoding Approach Distinguishes Hematopoietic Stem Cell Properties in Multiplexed In Vivo Experiments. Hum Gene Ther. 2021 10; 32(19-20):1280-1294. PMID: 34139894.
    Citations: 1     Fields:    Translation:AnimalsCells
  9. Klapdor R, Wang S, Morgan MA, Zimmermann K, Hachenberg J, Büning H, Dörk T, Hillemanns P, Schambach A. NK Cell-Mediated Eradication of Ovarian Cancer Cells with a Novel Chimeric Antigen Receptor Directed against CD44. Biomedicines. 2021 Sep 28; 9(10). PMID: 34680456.
    Citations: 1     
  10. Zhu S, Stanslowsky N, Fernández-Trillo J, Mamo TM, Yu P, Kalmbach N, Ritter B, Eggenschwiler R, Ouwendijk WJD, Mzinza D, Tan L, Leffler A, Spohn M, Brown RJP, Kropp KA, Kaever V, Ha TC, Narayanan P, Grundhoff A, Förster R, Schambach A, Verjans GMGM, Schmidt M, Kispert A, Cantz T, Gomis A, Wegner F, Viejo-Borbolla A. Generation of hiPSC-derived low threshold mechanoreceptors containing axonal termini resembling bulbous sensory nerve endings and expressing Piezo1 and Piezo2. Stem Cell Res. 2021 10; 56:102535. PMID: 34607262.
    Citations:    Fields:    Translation:HumansCells
  11. Büning H, Schambach A. A first step toward in vivo gene editing in patients. Nat Med. 2021 09; 27(9):1515-1517. PMID: 34462600.
    Citations:    Fields:    Translation:Humans
  12. Hassan JJ, Lieske A, Dörpmund N, Klatt D, Hoffmann D, Kleppa MJ, Kustikova OS, Stahlhut M, Schwarzer A, Schambach A, Maetzig T. A Multiplex CRISPR-Screen Identifies PLA2G4A as Prognostic Marker and Druggable Target for HOXA9 and MEIS1 Dependent AML. Int J Mol Sci. 2021 Aug 30; 22(17). PMID: 34502319.
    Citations:    Fields:    Translation:HumansCells
  13. Yang T, Poenisch M, Khanal R, Hu Q, Dai Z, Li R, Song G, Yuan Q, Yao Q, Shen X, Taubert R, Engel B, Jaeckel E, Vogel A, Falk CS, Schambach A, Gerovska D, Araúzo-Bravo MJ, Vondran FWR, Cantz T, Horscroft N, Balakrishnan A, Chevessier F, Ott M, Sharma AD. Therapeutic HNF4A mRNA attenuates liver fibrosis in a preclinical model. J Hepatol. 2021 12; 75(6):1420-1433. PMID: 34453962.
    Citations: 2     Fields:    Translation:Animals
  14. Schambach A, Morgan M, Fehse B. Two cases of T cell lymphoma following Piggybac-mediated CAR T cell therapy. Mol Ther. 2021 09 01; 29(9):2631-2633. PMID: 34433080.
    Citations: 2     Fields:    Translation:HumansCells
  15. Korf-Klingebiel M, Reboll MR, Polten F, Weber N, Jäckle F, Wu X, Kallikourdis M, Kunderfranco P, Condorelli G, Giannitsis E, Kustikova OS, Schambach A, Pich A, Widder JD, Bauersachs J, van den Heuvel J, Kraft T, Wang Y, Wollert KC. Myeloid-Derived Growth Factor Protects Against Pressure Overload-Induced Heart Failure by Preserving Sarco/Endoplasmic Reticulum Ca2+-ATPase Expression in Cardiomyocytes. Circulation. 2021 10 12; 144(15):1227-1240. PMID: 34372689.
    Citations:    Fields:    Translation:HumansAnimalsCells
  16. Morgan MA, Lange L, Schambach A. Targeted cytokine delivery: cell therapy to remodel the pre-metastatic niche. Signal Transduct Target Ther. 2021 07 25; 6(1):282. PMID: 34305139.
    Citations:    Fields:    Translation:HumansAnimals
  17. Janosz E, Hetzel M, Spielmann H, Tumpara S, Rossdam C, Schwabbauer M, Kloos D, von Kaisenberg C, Schambach A, Buettner FFR, Janciauskiene S, Lachmann N, Moritz T. Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo. Gene Ther. 2021 09; 28(9):477-493. PMID: 34276045.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  18. Morgan MA, Kloos A, Lenz D, Kattre N, Nowak J, Bentele M, Keisker M, Dahlke J, Zimmermann K, Sauer M, Heuser M, Schambach A. Improved Activity against Acute Myeloid Leukemia with Chimeric Antigen Receptor (CAR)-NK-92 Cells Designed to Target CD123. Viruses. 2021 07 14; 13(7). PMID: 34372571.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  19. Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U, Schambach A, Rothe M. Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning. Mol Ther. 2021 12 01; 29(12):3383-3397. PMID: 34174440.
    Citations:    Fields:    
  20. Dahlke J, Schott JW, Vollmer Barbosa P, Klatt D, Selich A, Lachmann N, Morgan M, Moritz T, Schambach A. Efficient Genetic Safety Switches for Future Application of iPSC-Derived Cell Transplants. J Pers Med. 2021 Jun 17; 11(6). PMID: 34204193.
    Citations: 1     
  21. Walcher L, Kistenmacher AK, Sommer C, Böhlen S, Ziemann C, Dehmel S, Braun A, Tretbar US, Klöß S, Schambach A, Morgan M, Löffler D, Kämpf C, Blumert C, Reiche K, Beckmann J, König U, Standfest B, Thoma M, Makert GR, Ulbert S, Kossatz-Böhlert U, Köhl U, Dünkel A, Fricke S. Low Energy Electron Irradiation Is a Potent Alternative to Gamma Irradiation for the Inactivation of (CAR-)NK-92 Cells in ATMP Manufacturing. Front Immunol. 2021; 12:684052. PMID: 34149724.
    Citations: 3     Fields:    Translation:HumansCells
  22. Franzen J, Georgomanolis T, Selich A, Kuo CC, Stöger R, Brant L, Mulabdic MS, Fernandez-Rebollo E, Grezella C, Ostrowska A, Begemann M, Nikolic M, Rath B, Ho AD, Rothe M, Schambach A, Papantonis A, Wagner W. DNA methylation changes during long-term in vitro cell culture are caused by epigenetic drift. Commun Biol. 2021 05 19; 4(1):598. PMID: 34011964.
    Citations: 2     Translation:HumansCells
  23. 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 EM, Malech HL, Thrasher AJ, Santilli G. Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease. Hum Gene Ther. 2021 09; 32(17-18):949-958. PMID: 33740872.
    Citations:    Fields:    Translation:HumansAnimals
  24. 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 04 29; 137(17):2326-2336. PMID: 33545713.
    Citations: 1     Fields:    Translation:AnimalsCells
  25. 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 08 04; 29(8):2535-2553. PMID: 33831558.
    Citations: 2     Fields:    Translation:HumansCells
  26. 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 09; 28(9):494-512. PMID: 33753908.
    Citations: 3     Fields:    Translation:HumansCells
  27. 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: 1     
  28. 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 03 05; 11(1):5294. PMID: 33674652.
    Citations:    Fields:    Translation:HumansAnimalsCells
  29. 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 May; 78(9):4143-4160. PMID: 33559689.
    Citations: 3     Fields:    Translation:HumansAnimalsCells
  30. Guerrouahen B, Elnaggar M, Al-Mohannadi A, Kizhakayil D, Bonini C, Benjamin R, Brentjens R, Buchholz CJ, Casorati G, Ferrone S, Locke FL, Martin F, Schambach A, Turtle C, Veys P, van der Vliet HJ, Maccalli C. Proceedings From the First International Workshop at Sidra Medicine: "Engineered Immune Cells in Cancer Immunotherapy (EICCI): From Discovery to Off-the-Shelf Development", 15th-16th February 2019, Doha, Qatar. Front Immunol. 2020; 11:589381. PMID: 33584653.
    Citations:    Fields:    Translation:HumansAnimalsCells
  31. 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 01; 32(1-2):77-95. PMID: 33023317.
    Citations: 3     Fields:    Translation:HumansCells
  32. 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: 1     
  33. 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. 2021 05; 32(9-10):458-472. PMID: 33012194.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  34. 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: 1     
  35. 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: 2     Fields:    Translation:HumansAnimalsCells
  36. 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:    Translation:HumansCells
  37. 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: 5     Fields:    Translation:Humans
  38. 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:    Translation:HumansAnimalsCells
  39. 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
  40. 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: 23     Fields:    Translation:HumansAnimalsCells
  41. 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: 3     Fields:    Translation:AnimalsCells
  42. 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: 1     Fields:    
  43. 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: 7     Fields:    Translation:HumansCellsCTClinical Trials
  44. 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: 4     Fields:    Translation:HumansAnimals
  45. 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: 7     Fields:    Translation:HumansAnimalsCells
  46. 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: 6     Fields:    Translation:HumansCells
  47. 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: 11     
  48. 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: 9     
  49. 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: 2     Fields:    Translation:HumansCells
  50. 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: 22     
  51. 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: 25     Fields:    Translation:HumansCells
  52. 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:    Translation:HumansAnimalsCells
  53. 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: 10     Fields:    Translation:HumansCells
  54. 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: 10     Fields:    
  55. 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: 10     
  56. 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: 8     Fields:    Translation:HumansAnimalsCells
  57. 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: 6     Fields:    Translation:HumansAnimalsCells
  58. 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: 9     Fields:    Translation:HumansCells
  59. 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: 7     
  60. 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
  61. 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: 2     Fields:    Translation:AnimalsCells
  62. 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: 4     Fields:    Translation:HumansAnimalsCells
  63. 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: 18     
  64. 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: 15     Fields:    Translation:HumansCells
  65. 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: 17     Fields:    Translation:HumansAnimalsCells
  66. 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: 21     Fields:    Translation:HumansCells
  67. 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: 36     Fields:    Translation:HumansAnimalsCells
  68. 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: 6     
  69. Morgan MA, Schambach A. Engineering CAR-T Cells for Improved Function Against Solid Tumors. Front Immunol. 2018; 9:2493. PMID: 30420856.
    Citations: 32     Fields:    Translation:HumansCells
  70. Philipp F, Selich A, Rothe M, Hoffmann D, Rittinghausen S, Morgan MA,