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Division Medical Biotechnology

Adney EM, Ochmann MT, Sil S, Truong DM, Mita P, Wang X, Kahler DJ, Fenyö D, Holt LJ, Boeke JD (2019): Comprehensive Scanning Mutagenesis of Human Retrotransposon LINE-1 Identifies Motifs Essential for Function.
Genetics 213: 1401-1414.
Online-Abstract

Agarwal S, Hanauer JDS, Frank AM, Riechert V, Thalheimer FB, Buchholz CJ (2020): In vivo generation of CAR T cells selectively in human CD4+ lymphocytes.
Mol Ther 28: 1783-1794.
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Ali S, Kjeken R, Niederlaender C, Markey G, Saunders TS, Opsata M, Moltu K, Bremnes B, Grønevik E, Muusse M, Håkonsen GD, Skibeli V, Kalland ME, Wang I, Buajordet I, Urbaniak A, Johnston J, Rantell K, Kerwash E, Schuessler-Lenz M, Salmonson T, Bergh J, Gisselbrecht C, Tzogani K, Papadouli I, Pignatti F (2020): The European Medicines Agency Review of Kymriah (Tisagenlecleucel) for the Treatment of Acute Lymphoblastic Leukemia and Diffuse Large B-Cell Lymphoma.
Oncologist 25: e321-e327.
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Amberger M, Ivics Z (2020): Latest Advances for the Sleeping Beauty Transposon System: 23 Years of Insomnia but Prettier than Ever: Refinement and Recent Innovations of the Sleeping Beauty Transposon System Enabling Novel, Nonviral Genetic Engineering Applications.
Bioessays 42: e2000136.
Online-Abstract

Ayyappan Jaguva Vasudevan A, Balakrishnan K, Gertzen CGW, Borveto F, Zhang Z, Sangwiman A, Held U, Küstermann C, Banerjee S, Schumann GG, Häussinger D, Bravo IG, Gohlke H, Münk C (2020): Loop 1 of APOBEC3C regulates its antiviral activity against HIV-1.
J Mol Biol 432: 6200-6227.
Online-Abstract

Baylis SA, Miskey C, Blümel J, Kaiser M, Kapusinszky B, Delwart E (2020): Identification of a Novel Bovine Copiparvovirus in Pooled Fetal Bovine Serum.
Virus Genes 56: 522-526.
Online-Abstract

Bittorf P, Bergmann T, Merlin S, Olgasi C, Pullig O, Sanzenbacher R, Zierau M, Walles H, Follenzi A, Braspenning J (2020): Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells.
Mol Ther Methods Clin Dev 18: 176-188.
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Börger V, Weiss DJ, Anderson JD, Borràs FE, Bussolati B, Carter DRF, Dominici M, Falcón-Pérez JM, Gimona M, Hill AF, Hoffman AM, de Kleijn D, Levine BL, Lim R, Lötvall J, Mitsialis A, Monguió-Tortajada M, Muraca M, Nieuwland R, Nowocin A, O'Driscoll L, Ortiz LA, Phinney DG, Reischl I, Rohde E, Sanzenbacher R, Théry C, Wei ST, Witwer KW, Lim SK, Giebel B (2020): ISEV and ISCT Statement on EVs From MSCs and Other Cells: Considerations for Potential Therapeutic Agents to Suppress COVID-19.
Cytotherapy 22: 482-485.
Online-Abstract

Brown RJP, Tegtmeyer B, Sheldon J, Khera T, Anggakusuma, Todt D, Vieyres G, Weller R, Joecks S, Zhang Y, Sake S, Bankwitz D, Welsch K, Ginkel C, Engelmann M, Gerold G, Steinmann E, Yuan Q, Ott M, Vondran FWR, Krey T, Ströh LJ, Miskey C, Ivics Z, Herder V, Baumgärtner W, Lauber C, Seifert M, Tarr AW, McClure CP, Randall G, Baktash Y, Ploss A, Thi VLD, Michailidis E, Saeed M, Verhoye L, Meuleman P, Goedecke N, Wirth D, Rice CM, Pietschmann T (2020): Liver-expressed Cd302 and Cr1l limit hepatitis C virus cross-species transmission to mice.
Sci Adv 6: eabd3233.
Online-Abstract

Cathomen T, Schüle S, Schüßler-Lenz M, Abou-El-Enein M (2019): The Human Genome Editing Race: Loosening Regulatory Standards for Commercial Advantage?.
Trends Biotechnol 37: 120-123.
Online-Abstract

Craig-Mueller N, Hammad R, Elling R, Alzubi J, Timm B, Kolter J, Knelangen N, Bednarski C, Gläser B, Ammann S, Ivics Z, Fischer J, Speckmann C, Schwarz K, Lachmann N, Ehl S, Moritz T, Henneke P, Cathomen T (2020): Modeling MyD88 Deficiency In Vitro Provides New Insights in Its Function.
Front Immunol 11: 608802.
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Cullmann K, Blokland KEC, Sebe A, Schenk F, Ivics Z, Heinz N, Modlich U (2019): Sustained and regulated gene expression by Tet-inducible "All-In-One" retroviral vectors containing the HNRPA2B1-CBX3 UCOE.
Biomaterials 192: 486-499.
Online-Abstract

Fintha A, Gasparics Á, Rosivall L, Sebe A (2019): Therapeutic Targeting of Fibrotic Epithelial-Mesenchymal Transition-An Outstanding Challenge.
Front Pharmacol 10: 388.
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Frank AM, Braun AH, Scheib L, Agarwal S, Schneider IC, Fusil F, Perian S, Sahin U, Thalheimer FB, Verhoeyen E, Buchholz CJ (2020): Combining T-cell-specific activation and in vivo gene delivery through CD3-targeted lentiviral vectors.
Blood Adv 4: 5702-5715.
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Frank AM, Buchholz CJ (2019): Surface-Engineered Lentiviral Vectors for Selective Gene Transfer into Subtypes of Lymphocytes.
Mol Ther Methods Clin Dev 12: 19-31.
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Frank AM, Weidner T, Brynza J, Uckert W, Buchholz CJ, Hartmann J (2020): CD8-Specific Designed Ankyrin Repeat Proteins Improve Selective Gene Delivery Into Human and Primate T Lymphocytes.
Hum Gene Ther 31: 679-691.
Online-Abstract

Gao B, Zong W, Miskey C, Ullah N, Diaby M, Chen C, Wang X, Ivics Z, Song C (2020): Intruder (DD38E), a recently evolved sibling family of DD34E/Tc1 transposons in animals.
Mob DNA 11: 32.
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Godehardt AW, Fischer N, Rauch P, Gulich B, Boller K, Church GM, Tönjes RR (2020): Characterization of porcine endogenous retrovirus particles released by the CRISPR/Cas9 inactivated cell line PK15 clone 15.
Xenotransplantation 27: e12563.
Online-Abstract

Godehardt AW, Ramm R, Gulich B, Tönjes RR, Hilfiker A (2020): Decellularized pig pulmonary heart valves-Depletion of nucleic acids measured by proviral PERV pol.
Xenotransplantation 27: e12565.
Online-Abstract

Godehardt AW, Tönjes RR (2020): Xenotransplantation of Decellularized Pig Heart valves-Regulatory Aspects in Europe.
Xenotransplantation 27: e12609.
Online-Abstract

Guan S, Munder A, Hedtfeld S, Braubach P, Glage S, Zhang L, Lienenklaus S, Schultze A, Hasenpusch G, Garrels W, Stanke F, Miskey C, Johler SM, Kumar Y, Tümmler B, Rudolph C, Ivics Z, Rosenecker J (2019): Self-assembled peptide-poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis.
Nat Nanotechnol 14: 287-297.
Online-Abstract

Hartmann J, Thalheimer FB, Höpfner F, Kerzel T, Khodosevich K, García-González D, Monyer H, Diester I, Büning H, Carette JE, Fries P, Buchholz CJ (2019): GluA4-Targeted AAV Vectors Deliver Genes Selectively to Interneurons while Relying on the AAV Receptor for Entry.
Mol Ther Methods Clin Dev 14: 252-260.
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Hawthorne WJ, Cowan PJ, Bühler LH, Yi S, Bottino R, Pierson RN, Ahn C, Azimzadeh A, Cozzi E, Gianello P, Lakey JRT, Luo M, Miyagawa S, Mohiuddin MM, Park CG, Schuurman HJ, Scobie L, Sykes M, Tector J, Tönjes RR, Wolf E, Nuñez JR, Wang W (2019): Third WHO Global Consultation on Regulatory Requirements for Xenotransplantation Clinical Trials, Changsha, Hunan, China December 12-14, 2018: "The 2018 Changsha Communiqué" The 10-Year Anniversary of The International Consultation on Xenotransplantation.
Xenotransplantation 26: e12513.
Online-Abstract

Hernandez M, Recalde S, Garcia-Garcia L, Bezunartea J, Miskey C, Johnen S, Diarra S, Sebe A, Rodriguez-Madoz JR, Pouillot S, Marie C, Izsvák Z, Scherman D, Kropp M, Prosper F, Thumann G, Ivics Z, Garcia-Layana A, Fernandez-Robredo P (2019): Preclinical Evaluation of a Cell-Based Gene Therapy Using the Sleeping Beauty Transposon System in Choroidal Neovascularization.
Mol Ther Methods Clin Dev 15: 403-417.
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Ivics Z, Amberger M, Zahn T, Hildt E (2020): Immuntherapien zur Behandlung der chronischen Hepatitis-B-Virusinfektion - eine Übersicht unter besonderer Berücksichtigung von CAR-T-Zellen.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 63: 1357-1364.
Online-Abstract

Jamali A, Kapitza L, Schaser T, Johnston ICD, Buchholz CJ, Hartmann J (2019): Highly Efficient and Selective CAR-Gene Transfer Using CD4- and CD8-Targeted Lentiviral Vectors.
Mol Ther Methods Clin Dev 13: 371-379.
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Kesselring L, Miskey C, Zuliani C, Querques I, Kapitonov V, Laukó A, Fehér A, Palazzo A, Diem T, Lustig J, Sebe A, Wang Y, Dinnyés A, Izsvák Z, Barabas O, Ivics Z (2020): A single amino acid switch converts the Sleeping Beauty transposase into an efficient unidirectional excisionase with utility in stem cell reprogramming.
Nucleic Acids Res 48: 316-331.
Online-Abstract

Kovač A, Miskey C, Menzel M, Grueso E, Gogol-Döring A, Ivics Z (2020): RNA-guided Retargeting of Sleeping Beauty Transposition in Human Cells.
Elife 9: e53868.
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Kurtz A, Elsallab M, Sanzenbacher R, Abou-El-Enein M (2019): Linking Scattered Stem Cell-Based Data to Advance Therapeutic Development.
Trends Mol Med 25: 8-19.
Online-Abstract

Laidou S, Alanis-Lobato G, Pribyl J, Raskó T, Tichy B, Mikulasek K, Tsagiopoulou M, Oppelt J, Kastrinaki G, Lefaki M, Singh M, Zink A, Chondrogianni N, Psomopoulos F, Prigione A, Ivics Z, Pospisoilova S, Skladal P, Izsvák Z, Andrade-Navarro MA, Petrakis S (2020): Nuclear Inclusions of Pathogenic ataxin-1 Induce Oxidative Stress and Perturb the Protein Synthesis Machinery.
Redox Biol 32: 101458.
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Lehmann F, Rau J, Malcolm B, Sander M, von Heymann C, Moormann T, Geyer T, Balzer F, Wernecke KD, Kaufner L (2019): Why does a point of care guided transfusion algorithm not improve blood loss and transfusion practice in patients undergoing high-risk cardiac surgery? A prospective randomized controlled pilot study.
BMC Anesthesiol 19: 24.
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Merker M, Wagner J, Kreyenberg H, Heim C, Moser LM, Wels WS, Bonig H, Ivics Z, Ullrich E, Klingebiel T, Bader P, Rettinger E (2020): ERBB2-CAR-Engineered Cytokine-Induced Killer Cells Exhibit Both CAR-Mediated and Innate Immunity Against High-Risk Rhabdomyosarcoma.
Front Immunol 11: 581468.
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Müller-Berghaus J, Sarac SB, Schüssler-Lenz M (2020): Kombinationstherapien in der Immunonkologie: Differenzierte regulatorische Herangehensweisen.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 63: 1388-1395.
Online-Abstract

Palazzo A, Lorusso P, Miskey C, Walisko O, Gerbino A, Marobbio CMT, Ivics Z, Marsano RM1 (2019): Transcriptionally promiscuous "blurry" promoters in Tc1/mariner transposons allow transcription in distantly related genomes.
Mob DNA 10: 13.
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Papadouli I, Mueller-Berghaus J, Beuneu C, Ali S, Hofner B, Petavy F, Tzogani K, Miermont A, Norga K, Kholmanskikh O, Leest T, Schuessler-Lenz M, Salmonson T, Gisselbrecht C, Garcia JL, Pignatti F (2020): EMA Review of Axicabtagene Ciloleucel (Yescarta) for the Treatment of Diffuse Large B-Cell Lymphoma.
Oncologist 25: 894-902.
Online-Abstract

Querques I, Mades A, Zuliani C, Miskey C, Alb M, Grueso E, Machwirth M, Rausch T, Einsele H, Ivics Z, Hudecek M, Barabas O (2019): A highly soluble Sleeping Beauty transposase improves control of gene insertion.
Nat Biotechnol 37: 1502-1512.
Online-Abstract

Reichart B, Längin M, Radan J, Mokelke M, Buttgereit I, Ying J, Fresch AK, Mayr T, Issl L, Buchholz S, Michel S, Ellgass R, Mihalj M, Egerer S, Baehr A, Kessler B, Kemter E, Kurome M, Zakhartchenko V, Steen S, Sjöberg T, Paskevicius A, Krüger L, Fiebig U, Denner J, Godehardt AW, Tönjes RR, Milusev A, Sfriso R, Rieben R, Walz C, Kirchner T, Ayares D, Lampe K, Schönmann U, Hagl C, Wolf E, Klymiuk N, Abicht JM, Brenner P (2020): Pig-to-non-human primate heart transplantation: the final step toward clinical xenotransplantation?.
J Heart Lung Transplant 39: 751-757.
Online-Abstract

Reul J, Muik A, Buchholz CJ (2019): Ligand Coupling to the AAV Capsid for Cell-Specific Gene Transfer.
Methods Mol Biol 1950: 35-50.
Online-Abstract

Sang Y, Gao B, Diaby M, Zong W, Chen C, Shen D, Wang S, Wang Y, Ivics Z, Song C (2019): Incomer, a DD36E family of Tc1/mariner transposons newly discovered in animals.
Mob DNA 10: 45.
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Sanzenbacher R, Frech M (2019): Streitpunkt Eigenfettbehandlung: Wenn Gewebe zur Arznei wird.
Dtsch Arztebl 116: A28-A29, A6.
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Savage AL, Ilera-Lopez A, Iacoangeli A, Bubb VJ, Smith B, Troakes C, Alahmady N, Koks S, Schumann GG, Al-Chalabi A, Quinn JP (2020): Frequency and methylation status of selected retrotransposition competent L1 loci in amyotrophic lateral sclerosis.
Mol Brain 13: 154.
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Savage AL, Schumann GG, Breen G, Bubb VJ, Al-Chalabi A, Quinn JP (2019): Retrotransposons in the development and progression of amyotrophic lateral sclerosis.
J Neurol Neurosurg Psychiatry 90: 284-293.
Online-Abstract

Scheuermann B, Diem T, Ivics Z, Andrade-Navarro MA (2019): Evolution-guided evaluation of the inverted terminal repeats of the synthetic transposon Sleeping Beauty.
Sci Rep 9: 1171.
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Schilling-Leiß D, Frech M, Tönjes RR, Günay-Winter D (2019): Fragen zur Transplantation von humanen Knochenpräparationen und Weichgewebe.
Implantologie 27: 383-385.
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Schuessler-Lenz M, Enzmann H, Vamvakas S (2020): Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia.
Clin Pharmacol Ther 107: 492-494.
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Schumann GG, Fuchs NV, Tristán-Ramos P, Sebe A, Ivics Z, Heras SR (2019): The impact of transposable element activity on therapeutically relevant human stem cells.
Mob DNA 10: 9.
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Sebe A, Anliker B, Rau J, Renner M (2020): Genetisch modifizierte regulatorische T-Zellen: Therapiekonzepte und ihr regulatorischer Rahmen.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 63: 1403-1411.
Online-Abstract

Shen D, Gao B, Miskey C, Chen C, Sang Y, Zong W, Wang S, Wang Y, Wang X, Ivics Z, Song C (2020): Multiple Invasions of Visitor, a DD41D Family of Tc1/mariner Transposons, throughout the Evolution of Vertebrates.
Genome Biol Evol 12: 1060-1073.
Online-Abstract

Singh M, Widmann TJ, Vikas Bansal, Cortes JL, Schumann GG, Wunderlich S, Martin U, Garcia-Canadas M, Garcia- Perez JL, Hurst LD, Izsvak Z (2019): The selection arena in early human blastocysts resolves the pluripotent inner cell mass.
bioRxiv Feb 4 [Epub ahead of print].
Online-Abstract