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Umbilical cord as a long-term source of activatable mesenchymal stromal cells for immunomodulation.

Selich A, Zimmermann K, Tenspolde M, Dittrich-Breiholz O, von Kaisenberg C, Schambach A, Rothe M.

Stem Cell Res Ther. 2019 Sep 23;10(1):285. doi: 10.1186/s13287-019-1376-9.


Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.

Klatt D, Cheng E, Philipp F, Selich A, Dahlke J, Schmidt RE, Schott JW, Büning H, Hoffmann D, Thrasher AJ, Schambach A.

Stem Cell Reports. 2019 Oct 8;13(4):590-598. doi: 10.1016/j.stemcr.2019.08.008. Epub 2019 Sep 19.


Chimeric antigen receptor-induced BCL11B suppression propagates NK-like cell development.

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.

J Clin Invest. 2019 Sep 3. pii: 126350. doi: 10.1172/JCI126350. [Epub ahead of print]


Capsid Engineering Overcomes Barriers Toward Adeno-Associated Virus Vector-Mediated Transduction of Endothelial Cells.

Zhang L, Rossi A, Lange L, Meumann N, Koitzsch U, Christie K, Nesbit MA, Moore CBT, Hacker UT, Morgan M, Hoffmann D, Zengel J, Carette JE, Schambach A, Salvetti A, Odenthal M, Büning H.

Hum Gene Ther. 2019 Oct;30(10):1284-1296. doi: 10.1089/hum.2019.027.


Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application.

Schott JW, León-Rico D, Ferreira CB, Buckland KF, Santilli G, Armant MA, Schambach A, Cavazza A, Thrasher AJ.

Mol Ther Methods Clin Dev. 2019 Jun 7;14:134-147. doi: 10.1016/j.omtm.2019.05.015. eCollection 2019 Sep 13.


Cytokine Selection of MSC Clones with Different Functionality.

Selich A, Ha TC, Morgan M, Falk CS, von Kaisenberg C, Schambach A, Rothe M.

Stem Cell Reports. 2019 Aug 13;13(2):262-273. doi: 10.1016/j.stemcr.2019.06.001. Epub 2019 Jul 11.


Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.

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.

Haematologica. 2019 Jul 9. pii: haematol.2018.214866. doi: 10.3324/haematol.2018.214866. [Epub ahead of print]


Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice.

Löfvall H, Rothe M, Schambach A, Henriksen K, Richter J, Moscatelli I.

Hum Gene Ther. 2019 Jul 3. doi: 10.1089/hum.2019.047. [Epub ahead of print]


Development of Automated Separation, Expansion, and Quality Control Protocols for Clinical-Scale Manufacturing of Primary Human NK Cells and Alpharetroviral Chimeric Antigen Receptor Engineering.

Oberschmidt O, Morgan M, Huppert V, Kessler J, Gardlowski T, Matthies N, Aleksandrova K, Arseniev L, Schambach A, Koehl U, Kloess S.

Hum Gene Ther Methods. 2019 Jun;30(3):102-120. doi: 10.1089/hgtb.2019.039. Epub 2019 May 16.


Preclinical Assessment of Suitable Natural Killer Cell Sources for Chimeric Antigen Receptor Natural Killer-Based "Off-the-Shelf" Acute Myeloid Leukemia Immunotherapies.

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.

Hum Gene Ther. 2019 Apr;30(4):381-401. doi: 10.1089/hum.2018.247. Epub 2019 Mar 18.


Characterization of a Novel Third-Generation Anti-CD24-CAR against Ovarian Cancer.

Klapdor R, Wang S, Morgan M, Dörk T, Hacker U, Hillemanns P, Büning H, Schambach A.

Int J Mol Sci. 2019 Feb 3;20(3). pii: E660. doi: 10.3390/ijms20030660.


Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.

Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014. eCollection 2018 Dec 14.


Bioreactor-based mass production of human iPSC-derived macrophages enables immunotherapies against bacterial airway infections.

Ackermann M, Kempf H, Hetzel M, Hesse C, Hashtchin AR, Brinkert K, Schott JW, Haake K, Kühnel MP, Glage S, Figueiredo C, Jonigk D, Sewald K, Schambach A, Wronski S, Moritz T, Martin U, Zweigerdt R, Munder A, Lachmann N.

Nat Commun. 2018 Nov 30;9(1):5088. doi: 10.1038/s41467-018-07570-7.


Engineering CAR-T Cells for Improved Function Against Solid Tumors.

Morgan MA, Schambach A.

Front Immunol. 2018 Oct 29;9:2493. doi: 10.3389/fimmu.2018.02493. eCollection 2018. Review.


Human Teratoma-Derived Hematopoiesis Is a Highly Polyclonal Process Supported by Human Umbilical Vein Endothelial Cells.

Philipp F, Selich A, Rothe M, Hoffmann D, Rittinghausen S, Morgan MA, Klatt D, Glage S, Lienenklaus S, Neuhaus V, Sewald K, Braun A, Schambach A.

Stem Cell Reports. 2018 Nov 13;11(5):1051-1060. doi: 10.1016/j.stemcr.2018.09.010. Epub 2018 Oct 18.


Transient Retrovirus-Based CRISPR/Cas9 All-in-One Particles for Efficient, Targeted Gene Knockout.

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.

Mol Ther Nucleic Acids. 2018 Dec 7;13:256-274. doi: 10.1016/j.omtn.2018.09.006. Epub 2018 Sep 13.


CRISPR/Cas9 Immunoengineering of Hoxb8-Immortalized Progenitor Cells for Revealing CCR7-Mediated Dendritic Cell Signaling and Migration Mechanisms in vivo.

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.

Front Immunol. 2018 Aug 28;9:1949. doi: 10.3389/fimmu.2018.01949. eCollection 2018.


Chimeric Antigen Receptor T Cells: Extending Translation from Liquid to Solid Tumors.

Morgan MA, Schambach A.

Hum Gene Ther. 2018 Oct;29(10):1083-1097. doi: 10.1089/hum.2017.251. Epub 2018 Aug 29. Review.


Erratum: EVI1 carboxy-terminal phosphorylation is ATM-mediated and sustains transcriptional modulation and self-renewal via enhanced CtBP1 association.

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.

Nucleic Acids Res. 2018 Sep 19;46(16):8663. doi: 10.1093/nar/gky711. No abstract available.


Fluorescent genetic barcoding for cellular multiplex analyses.

Maetzig T, Morgan M, Schambach A.

Exp Hematol. 2018 Nov;67:10-17. doi: 10.1016/j.exphem.2018.08.001. Epub 2018 Aug 8. Review.


A patient-specific induced pluripotent stem cell model for West syndrome caused by ST3GAL3 deficiency.

van Diepen L, Buettner FFR, Hoffmann D, Thiesler CT, von Bohlen Und Halbach O, von Bohlen Und Halbach V, Jensen LR, Steinemann D, Edvardson S, Elpeleg O, Schambach A, Gerardy-Schahn R, Kuss AW.

Eur J Hum Genet. 2018 Dec;26(12):1773-1783. doi: 10.1038/s41431-018-0220-5. Epub 2018 Aug 8.


Effect of TP53 contact and conformational mutations on cell survival and erythropoiesis of human hematopoietic stem cells in a long term culture model.

Salari A, Thomay K, Lentes J, Ebersold J, Hagedorn M, Skawran B, Davenport C, Schambach A, Schlegelberger B, Göhring G.

Oncotarget. 2018 Jul 6;9(52):29869-29876. doi: 10.18632/oncotarget.25581. eCollection 2018 Jul 6.


EVI1 carboxy-terminal phosphorylation is ATM-mediated and sustains transcriptional modulation and self-renewal via enhanced CtBP1 association.

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.

Nucleic Acids Res. 2018 Sep 6;46(15):7662-7674. doi: 10.1093/nar/gky536. Erratum in: Nucleic Acids Res. 2018 Sep 19;46(16):8663.


Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

Poletti V, Charrier S, Corre G, Gjata B, Vignaud A, Zhang F, Rothe M, Schambach A, Gaspar HB, Thrasher AJ, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Mar 10;9:257-269. doi: 10.1016/j.omtm.2018.03.002. eCollection 2018 Jun 15.


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

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

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


T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.

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.

J Allergy Clin Immunol. 2018 Sep;142(3):904-913.e3. doi: 10.1016/j.jaci.2017.11.050. Epub 2018 Jan 31.


Pooled Generation of Lentiviral Tetracycline-Regulated microRNA Embedded Short Hairpin RNA Libraries.

Adams FF, Hoffmann T, Zuber J, Heckl D, Schambach A, Schwarzer A.

Hum Gene Ther Methods. 2018 Feb;29(1):16-29. doi: 10.1089/hgtb.2017.182.


Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells.

Pittermann E, Lachmann N, MacLean G, Emmrich S, Ackermann M, Göhring G, Schlegelberger B, Welte K, Schambach A, Heckl D, Orkin SH, Cantz T, Klusmann JH.

Blood Adv. 2017 Jun 2;1(14):903-914. doi: 10.1182/bloodadvances.2016003798. eCollection 2017 Jun 13.


Refined sgRNA efficacy prediction improves large- and small-scale CRISPR-Cas9 applications.

Labuhn M, Adams FF, Ng M, Knoess S, Schambach A, Charpentier EM, Schwarzer A, Mateo JL, Klusmann JH, Heckl D.

Nucleic Acids Res. 2018 Feb 16;46(3):1375-1385. doi: 10.1093/nar/gkx1268.


Impaired IFNγ-Signaling and Mycobacterial Clearance in IFNγR1-Deficient Human iPSC-Derived Macrophages.

Neehus AL, Lam J, Haake K, Merkert S, Schmidt N, Mucci A, Ackermann M, Schubert M, Happle C, Kühnel MP, Blank P, Philipp F, Goethe R, Jonigk D, Martin U, Kalinke U, Baumann U, Schambach A, Roesler J, Lachmann N.

Stem Cell Reports. 2018 Jan 9;10(1):7-16. doi: 10.1016/j.stemcr.2017.11.011. Epub 2017 Dec 14.


Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections.

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.

Blood. 2018 Feb 1;131(5):533-545. doi: 10.1182/blood-2017-10-812859. Epub 2017 Dec 12.


Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy.

Biasco L, Rothe M, Büning H, Schambach A.

Mol Ther Methods Clin Dev. 2017 Oct 5;8:21-30. doi: 10.1016/j.omtm.2017.10.002. eCollection 2018 Mar 16. Review.


Uncoupling the Oncogenic Engine.

Schambach A, Schott JW, Morgan MA.

Cancer Res. 2017 Nov 15;77(22):6060-6064. doi: 10.1158/0008-5472.CAN-17-2362. Epub 2017 Nov 2. Review.


Correction to: Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells.

Geis FK, Galla M, Hoffmann D, Kuehle J, Zychlinski D, Maetzig T, Schott JW, Schwarzer A, Goffinet C, Goff SP, Schambach A.

Retrovirology. 2017 Oct 18;14(1):48. doi: 10.1186/s12977-017-0372-3.


An RNA-targeted therapy for dystrophic epidermolysis bullosa.

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.

Nucleic Acids Res. 2017 Sep 29;45(17):10259-10269. doi: 10.1093/nar/gkx669.


Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis.

Biasco L, Rothe M, Schott JW, Schambach A.

Hematol Oncol Clin North Am. 2017 Oct;31(5):737-752. doi: 10.1016/j.hoc.2017.06.009. Review.


Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency.

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.

Hum Gene Ther Methods. 2017 Dec;28(6):318-329. doi: 10.1089/hgtb.2017.092. Epub 2017 Aug 30.


Improved Killing of Ovarian Cancer Stem Cells by Combining a Novel Chimeric Antigen Receptor-Based Immunotherapy and Chemotherapy.

Klapdor R, Wang S, Hacker U, Büning H, Morgan M, Dörk T, Hillemanns P, Schambach A.

Hum Gene Ther. 2017 Oct;28(10):886-896. doi: 10.1089/hum.2017.168. Epub 2017 Aug 24.


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.

Klöß S, Oberschmidt O, Morgan M, Dahlke J, Arseniev L, Huppert V, Granzin M, Gardlowski T, Matthies N, Soltenborn S, Schambach A, Koehl U.

Hum Gene Ther. 2017 Oct;28(10):897-913. doi: 10.1089/hum.2017.157. Epub 2017 Aug 15.


The CpG-sites of the CBX3 ubiquitous chromatin opening element are critical structural determinants for the anti-silencing function.

Kunkiel J, Gödecke N, Ackermann M, Hoffmann D, Schambach A, Lachmann N, Wirth D, Moritz T.

Sci Rep. 2017 Aug 11;7(1):7919. doi: 10.1038/s41598-017-04212-8.


The non-coding RNA landscape of human hematopoiesis and leukemia.

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.

Nat Commun. 2017 Aug 9;8(1):218. doi: 10.1038/s41467-017-00212-4.


Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis.

Moscatelli I, Löfvall H, Schneider Thudium C, Rothe M, Montano C, Kertész Z, Sirin M, Schulz A, Schambach A, Henriksen K, Richter J.

Hum Gene Ther. 2018 Aug;29(8):938-949. doi: 10.1089/hum.2017.053. Epub 2017 Oct 3.


Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration.

Stahlhut M, Schambach A, Kustikova OS.

Hum Gene Ther Methods. 2017 Aug;28(4):191-204. doi: 10.1089/hgtb.2017.073. Epub 2017 Jul 5.


Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease.

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.

Mol Ther Methods Clin Dev. 2017 May 12;5:241-258. doi: 10.1016/j.omtm.2017.05.003. eCollection 2017 Jun 16.


An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries.

Adams FF, Heckl D, Hoffmann T, Talbot SR, Kloos A, Thol F, Heuser M, Zuber J, Schambach A, Schwarzer A.

Biomaterials. 2017 Sep;139:102-115. doi: 10.1016/j.biomaterials.2017.05.032. Epub 2017 May 23.


Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells.

Geis FK, Galla M, Hoffmann D, Kuehle J, Zychlinski D, Maetzig T, Schott JW, Schwarzer A, Goffinet C, Goff SP, Schambach A.

Retrovirology. 2017 May 31;14(1):34. doi: 10.1186/s12977-017-0358-1. Erratum in: Retrovirology. 2017 Oct 18;14(1):48.


Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.

Vink CA, Counsell JR, Perocheau DP, Karda R, Buckley SMK, Brugman MH, Galla M, Schambach A, McKay TR, Waddington SN, Howe SJ.

Mol Ther. 2017 Aug 2;25(8):1790-1804. doi: 10.1016/j.ymthe.2017.04.028. Epub 2017 May 24.


Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.

Debnath S, Jaako P, Siva K, Rothe M, Chen J, Dahl M, Gaspar HB, Flygare J, Schambach A, Karlsson S.

Mol Ther. 2017 Aug 2;25(8):1805-1814. doi: 10.1016/j.ymthe.2017.04.002. Epub 2017 Apr 20.


Scavenger receptor class B member 1 (SCARB1) variants modulate hepatitis C virus replication cycle and viral load.

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.

J Hepatol. 2017 Aug;67(2):237-245. doi: 10.1016/j.jhep.2017.03.020. Epub 2017 Mar 29.


Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells.

Hoffmann D, Schott JW, Geis FK, Lange L, Müller FJ, Lenz D, Zychlinski D, Steinemann D, Morgan M, Moritz T, Schambach A.

Gene Ther. 2017 May;24(5):298-307. doi: 10.1038/gt.2017.20. Epub 2017 Apr 20.


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