My Bibliography

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Tasfaout H, Halbert CL, McMillen TS, Allen JM, Reyes TR, Flint GV, Grimm D, Hauschka SD, Regnier M, Chamberlain JS. Split intein-mediated protein trans-splicing to express large dystrophins. Nature. 2024 Aug;632(8023):192-200. doi: 10.1038/s41586-024-07710-8. Epub 2024 Jul 17. PubMed PMID: 39020181; PubMed Central PMCID: PMC11335042.

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Bubeck F, Grimm D. When size matters: A novel compact Cas12a variant for in vivo genome editing. PLoS Biol. 2024 Jul;22(7):e3002637. doi: 10.1371/journal.pbio.3002637. eCollection 2024 Jul. PubMed PMID: 39018342; PubMed Central PMCID: PMC11253977.

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Wagner JT, Müller-Schmucker SM, Wang W, Arnold P, Uhlig N, Issmail L, Eberlein V, Damm D, Roshanbinfar K, Ensser A, Oltmanns F, Peter AS, Temchura V, Schrödel S, Engel FB, Thirion C, Grunwald T, Wuhrer M, Grimm D, Überla K. Influence of AAV vector tropism on long-term expression and Fc-γ receptor binding of an antibody targeting SARS-CoV-2. Commun Biol. 2024 Jul 16;7(1):865. doi: 10.1038/s42003-024-06529-3. PubMed PMID: 39009807; PubMed Central PMCID: PMC11250830.

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Muñoz S, Bertolin J, Jimenez V, Jaén ML, Garcia M, Pujol A, Vilà L, Sacristan V, Barbon E, Ronzitti G, El Andari J, Tulalamba W, Pham QH, Ruberte J, VandenDriessche T, Chuah MK, Grimm D, Mingozzi F, Bosch F. Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy. Mol Metab. 2024 Mar;81:101899. doi: 10.1016/j.molmet.2024.101899. Epub 2024 Feb 10. PubMed PMID: 38346589; PubMed Central PMCID: PMC10877955.

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Schiller L, Ko C, Kosinska AD, Grimm D, Protzer U. Production and Purification of Adeno-Associated Viral Vectors for the Development of Immune-Competent Mouse Models of Persistent Hepatitis B Virus Replication. Methods Mol Biol. 2024;2837:207-218. doi: 10.1007/978-1-0716-4027-2_18. PubMed PMID: 39044087.

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Theuerkauf SA, Herrera-Carrillo E, John F, Zinser LJ, Molina MA, Riechert V, Thalheimer FB, Börner K, Grimm D, Chlanda P, Berkhout B, Buchholz CJ. AAV vectors displaying bispecific DARPins enable dual-control targeted gene delivery. Biomaterials. 2023 Dec;303:122399. doi: 10.1016/j.biomaterials.2023.122399. Epub 2023 Nov 16. PubMed PMID: 37992599; PubMed Central PMCID: PMC10721713.

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Thürkauf M, Lin S, Oliveri F, Grimm D, Platt RJ, Rüegg MA. Fast, multiplexable and efficient somatic gene deletions in adult mouse skeletal muscle fibers using AAV-CRISPR/Cas9. Nat Commun. 2023 Sep 30;14(1):6116. doi: 10.1038/s41467-023-41769-7. PubMed PMID: 37777530; PubMed Central PMCID: PMC10542775.

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Moreno Velasquez SD, Gerstmann E, Grimm D. Goody two plasmids: An optimized transient transfection system for AAV vector production. Mol Ther Methods Clin Dev. 2023 Sep 14;30:191-193. doi: 10.1016/j.omtm.2023.06.010. eCollection 2023 Sep 14. PubMed PMID: 37519408; PubMed Central PMCID: PMC10371777.

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Stajković N, Liu Y, Arsić A, Meng N, Lyu H, Zhang N, Grimm D, Lerche H, Nikić-Spiegel I. Direct fluorescent labeling of NF186 and NaV1.6 in living primary neurons using bioorthogonal click chemistry. J Cell Sci. 2023 Jun 15;136(12). doi: 10.1242/jcs.260600. Epub 2023 Jun 28. PubMed PMID: 37288813; PubMed Central PMCID: PMC10323244.

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Grosch M, Schraft L, Chan A, Küchenhoff L, Rapti K, Ferreira AM, Kornienko J, Li S, Radke MH, Krämer C, Clauder-Münster S, Perlas E, Backs J, Gotthardt M, Dieterich C, van den Hoogenhof MMG, Grimm D, Steinmetz LM. Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy. Nat Commun. 2023 Jun 22;14(1):3714. doi: 10.1038/s41467-023-39352-1. PubMed PMID: 37349314; PubMed Central PMCID: PMC10287752.

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Grimm D, Nonnenmacher M. Every little bit helps: A single-residue switch in a vascular AAV enables blood-brain barrier penetration. Mol Ther Methods Clin Dev. 2023 Jun 8;29:364-365. doi: 10.1016/j.omtm.2023.04.007. eCollection 2023 Jun 8. PubMed PMID: 37214312; PubMed Central PMCID: PMC10199398.

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Liu J, Koay TW, Maiakovska O, Zayas M, Grimm D. Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors. Hum Gene Ther. 2023 May;34(9-10):350-364. doi: 10.1089/hum.2023.057. Review. PubMed PMID: 37082964.

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Kermani F, Mosqueira M, Peters K, Lemma ED, Rapti K, Grimm D, Bastmeyer M, Laugsch M, Hecker M, Ullrich ND. Membrane remodelling triggers maturation of excitation-contraction coupling in 3D-shaped human-induced pluripotent stem cell-derived cardiomyocytes. Basic Res Cardiol. 2023 Mar 29;118(1):13. doi: 10.1007/s00395-023-00984-5. PubMed PMID: 36988697; PubMed Central PMCID: PMC10060306.

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Szumska J, Grimm D. Boosters for adeno-associated virus (AAV) vector (r)evolution. Cytotherapy. 2023 Mar;25(3):254-260. doi: 10.1016/j.jcyt.2022.07.005. Epub 2022 Aug 20. PubMed PMID: 35999132.

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Yang JH, Hayano M, Griffin PT, Amorim JA, Bonkowski MS, Apostolides JK, Salfati EL, Blanchette M, Munding EM, Bhakta M, Chew YC, Guo W, Yang X, Maybury-Lewis S, Tian X, Ross JM, Coppotelli G, Meer MV, Rogers-Hammond R, Vera DL, Lu YR, Pippin JW, Creswell ML, Dou Z, Xu C, Mitchell SJ, Das A, O'Connell BL, Thakur S, Kane AE, Su Q, Mohri Y, Nishimura EK, Schaevitz L, Garg N, Balta AM, Rego MA, Gregory-Ksander M, Jakobs TC, Zhong L, Wakimoto H, El Andari J, Grimm D, Mostoslavsky R, Wagers AJ, Tsubota K, Bonasera SJ, Palmeira CM, Seidman JG, Seidman CE, Wolf NS, Kreiling JA, Sedivy JM, Murphy GF, Green RE, Garcia BA, Berger SL, Oberdoerffer P, Shankland SJ, Gladyshev VN, Ksander BR, Pfenning AR, Rajman LA, Sinclair DA. Loss of epigenetic information as a cause of mammalian aging. Cell. 2023 Jan 19;186(2):305-326.e27. doi: 10.1016/j.cell.2022.12.027. Epub 2023 Jan 12. PubMed PMID: 36638792; PubMed Central PMCID: PMC10166133.

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Maiakovska O, Baumgartl C, Grimm D. Determination of AAV properties by single amino acids: Go(o)d is in the details. Mol Ther Methods Clin Dev. 2022 Dec 8;27:93-95. doi: 10.1016/j.omtm.2022.09.006. eCollection 2022 Dec 8. PubMed PMID: 36212908; PubMed Central PMCID: PMC9529493.

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Waithaka A, Maiakovska O, Grimm D, do Nascimento LM, Clayton C. Sequences and proteins that influence mRNA processing in Trypanosoma brucei: Evolutionary conservation of SR-domain and PTB protein functions. PLoS Negl Trop Dis. 2022 Oct;16(10):e0010876. doi: 10.1371/journal.pntd.0010876. eCollection 2022 Oct. PubMed PMID: 36288402; PubMed Central PMCID: PMC9639853.

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Rapti K, Maiakovska O, Becker J, Szumska J, Zayas M, Bubeck F, Liu J, Gerstmann E, Krämer C, Wiedtke E, Grimm D. Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants. J Vis Exp. 2022 Oct 18;(188). doi: 10.3791/64389. PubMed PMID: 36342179.

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Gadenstaetter AJ, Schmutzler L, Grimm D, Landegger LD. Intranasal application of adeno-associated viruses: a systematic review. Transl Res. 2022 Oct;248:87-110. doi: 10.1016/j.trsl.2022.05.002. Epub 2022 May 18. Review. PubMed PMID: 35597541.

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El Andari J, Renaud-Gabardos E, Tulalamba W, Weinmann J, Mangin L, Pham QH, Hille S, Bennett A, Attebi E, Bourges E, Leborgne C, Guerchet N, Fakhiri J, Krämer C, Wiedtke E, McKenna R, Guianvarc'h L, Toueille M, Ronzitti G, Hebben M, Mingozzi F, VandenDriessche T, Agbandje-McKenna M, Müller OJ, Chuah MK, Buj-Bello A, Grimm D. Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders. Sci Adv. 2022 Sep 23;8(38):eabn4704. doi: 10.1126/sciadv.abn4704. Epub 2022 Sep 21. PubMed PMID: 36129972; PubMed Central PMCID: PMC9491714.

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Rovai A, Chung B, Hu Q, Hook S, Yuan Q, Kempf T, Schmidt F, Grimm D, Talbot SR, Steinbrück L, Götting J, Bohne J, Krooss SA, Ott M. In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice. Nat Commun. 2022 Sep 5;13(1):5215. doi: 10.1038/s41467-022-32906-9. PubMed PMID: 36064805; PubMed Central PMCID: PMC9445023.

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Becker J, Fischer N, Grimm D. Right on target: The next class of efficient, safe, and specific RNAi triggers. Mol Ther Nucleic Acids. 2022 Jun 14;28:363-365. doi: 10.1016/j.omtn.2022.03.027. eCollection 2022 Jun 14. PubMed PMID: 35505965; PubMed Central PMCID: PMC9035382.

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Flitsch LJ, Börner K, Stüllein C, Ziegler S, Sonntag-Buck V, Wiedtke E, Semkova V, Au Yeung SWC, Schlee J, Hajo M, Mathews M, Ludwig BS, Kossatz S, Kessler H, Grimm D, Brüstle O. Identification of adeno-associated virus variants for gene transfer into human neural cell types by parallel capsid screening. Sci Rep. 2022 May 19;12(1):8356. doi: 10.1038/s41598-022-12404-0. PubMed PMID: 35589936; PubMed Central PMCID: PMC9120183.

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Becker J, Stanifer ML, Leist SR, Stolp B, Maiakovska O, West A, Wiedtke E, Börner K, Ghanem A, Ambiel I, Tse LV, Fackler OT, Baric RS, Boulant S, Grimm D. Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors. Mol Ther. 2022 May 4;30(5):2005-2023. doi: 10.1016/j.ymthe.2022.01.024. Epub 2022 Jan 14. PubMed PMID: 35038579; PubMed Central PMCID: PMC8758558.

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Bauer A, Puglisi M, Nagl D, Schick JA, Werner T, Klingl A, El Andari J, Hornung V, Kessler H, Götz M, Grimm D, Brack-Werner R. Molecular Signature of Astrocytes for Gene Delivery by the Synthetic Adeno-Associated Viral Vector rAAV9P1. Adv Sci (Weinh). 2022 May;9(16):e2104979. doi: 10.1002/advs.202104979. Epub 2022 Apr 10. PubMed PMID: 35398994; PubMed Central PMCID: PMC9165502.

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Melendez E, Chondronasiou D, Mosteiro L, Martínez de Villarreal J, Fernández-Alfara M, Lynch CJ, Grimm D, Real FX, Alcamí J, Climent N, Pietrocola F, Serrano M. Natural killer cells act as an extrinsic barrier for in vivo reprogramming. Development. 2022 Apr 15;149(8). doi: 10.1242/dev.200361. Epub 2022 Apr 22. PubMed PMID: 35420133; PubMed Central PMCID: PMC9124575.

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Zhang C, Freistaedter A, Schmelas C, Gunkel M, Dao Thi VL, Grimm D. An RNA Interference/Adeno-Associated Virus Vector-Based Combinatorial Gene Therapy Approach Against Hepatitis E Virus. Hepatol Commun. 2022 Apr;6(4):878-888. doi: 10.1002/hep4.1842. Epub 2021 Oct 31. PubMed PMID: 34719133; PubMed Central PMCID: PMC8948557.

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Ernst L, Steinfeld B, Barayeu U, Klintzsch T, Kurth M, Grimm D, Dick TP, Rebelein JG, Bischofs IB, Keppler F. Methane formation driven by reactive oxygen species across all living organisms. Nature. 2022 Mar;603(7901):482-487. doi: 10.1038/s41586-022-04511-9. Epub 2022 Mar 9. PubMed PMID: 35264795.

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Maurer L, El Andari J, Rapti K, Spreyer L, Steinmann E, Grimm D, Dao Thi VL. Induction of Hepatitis E Virus Anti-ORF3 Antibodies from Systemic Administration of a Muscle-Specific Adeno-Associated Virus (AAV) Vector. Viruses. 2022 Jan 27;14(2). doi: 10.3390/v14020266. PubMed PMID: 35215859; PubMed Central PMCID: PMC8878420.

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Fakhiri J, Landegger LD, Grimm D. Breaking the sound barrier: Towards next-generation AAV vectors for gene therapy of hearing disorders. Hear Res. 2022 Jan;413:108092. doi: 10.1016/j.heares.2020.108092. Epub 2020 Oct 15. Review. PubMed PMID: 33268240.

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Mann AM, Schäfer W, Adriouch S, Börner K, Grimm D, Braren I, Koch-Nolte F. Enhanced Transduction of P2X7-Expressing Cells with Recombinant rAAV Vectors. Methods Mol Biol. 2022;2510:129-144. doi: 10.1007/978-1-0716-2384-8_7. PubMed PMID: 35776323.

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Kremer LPM, Cerrizuela S, Dehler S, Stiehl T, Weinmann J, Abendroth H, Kleber S, Laure A, El Andari J, Anders S, Marciniak-Czochra A, Grimm D, Martin-Villalba A. High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone. Mol Ther Methods Clin Dev. 2021 Dec 10;23:33-50. doi: 10.1016/j.omtm.2021.07.001. eCollection 2021 Dec 10. PubMed PMID: 34553001; PubMed Central PMCID: PMC8427210.

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Cheng M, Dietz L, Gong Y, Eichler F, Nammour J, Ng C, Grimm D, Maguire CA. Neutralizing Antibody Evasion and Transduction with Purified Extracellular Vesicle-Enveloped Adeno-Associated Virus Vectors. Hum Gene Ther. 2021 Dec;32(23-24):1457-1470. doi: 10.1089/hum.2021.122. PubMed PMID: 34445894; PubMed Central PMCID: PMC8742275.

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Fakhiri J, Grimm D. Best of most possible worlds: Hybrid gene therapy vectors based on parvoviruses and heterologous viruses. Mol Ther. 2021 Dec 1;29(12):3359-3382. doi: 10.1016/j.ymthe.2021.04.005. Epub 2021 Apr 5. Review. PubMed PMID: 33831556; PubMed Central PMCID: PMC8636155.

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Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7. PubMed PMID: 34819506; PubMed Central PMCID: PMC8613397.

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Marichal-Gallardo P, Börner K, Pieler MM, Sonntag-Buck V, Obr M, Bejarano D, Wolff MW, Kräusslich HG, Reichl U, Grimm D. Single-Use Capture Purification of Adeno-Associated Viral Gene Transfer Vectors by Membrane-Based Steric Exclusion Chromatography. Hum Gene Ther. 2021 Sep;32(17-18):959-974. doi: 10.1089/hum.2019.284. Epub 2021 Mar 30. PubMed PMID: 33554722; PubMed Central PMCID: PMC10116406.

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Luck R, Karakatsani A, Shah B, Schermann G, Adler H, Kupke J, Tisch N, Jeong HW, Back MK, Hetsch F, D'Errico A, De Palma M, Wiedtke E, Grimm D, Acker-Palmer A, von Engelhardt J, Adams RH, Augustin HG, Ruiz de Almodóvar C. The angiopoietin-Tie2 pathway regulates Purkinje cell dendritic morphogenesis in a cell-autonomous manner. Cell Rep. 2021 Aug 17;36(7):109522. doi: 10.1016/j.celrep.2021.109522. PubMed PMID: 34407407; PubMed Central PMCID: PMC9110807.

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Meng N, Grimm D. Membrane-destabilizing ionizable phospholipids: Novel components for organ-selective mRNA delivery and CRISPR-Cas gene editing. Signal Transduct Target Ther. 2021 May 25;6(1):206. doi: 10.1038/s41392-021-00642-z. PubMed PMID: 34035211; PubMed Central PMCID: PMC8149719.

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Hoffmann MD, Mathony J, Upmeier Zu Belzen J, Harteveld Z, Aschenbrenner S, Stengl C, Grimm D, Correia BE, Eils R, Niopek D. Optogenetic control of Neisseria meningitidis Cas9 genome editing using an engineered, light-switchable anti-CRISPR protein. Nucleic Acids Res. 2021 Mar 18;49(5):e29. doi: 10.1093/nar/gkaa1198. PubMed PMID: 33330940; PubMed Central PMCID: PMC7969004.

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Murenu E, Pavlou M, Richter L, Rapti K, Just S, Cehajic-Kapetanovic J, Tafrishi N, Hayes A, Scholey R, Lucas R, Büning H, Grimm D, Michalakis S. A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting. Mol Ther Methods Clin Dev. 2021 Mar 12;20:587-600. doi: 10.1016/j.omtm.2021.01.011. eCollection 2021 Mar 12. PubMed PMID: 33665228; PubMed Central PMCID: PMC7895692.

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Medert R, Jungmann A, Hildebrand S, Busch M, Grimm D, Flockerzi V, Müller OJ, Most P, Schumacher D, Freichel M. Development of an AAV9-RNAi-mediated silencing strategy to abrogate TRPM4 expression in the adult heart. Pflugers Arch. 2021 Mar;473(3):533-546. doi: 10.1007/s00424-021-02521-6. Epub 2021 Feb 13. PubMed PMID: 33580817; PubMed Central PMCID: PMC7940300.

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Yu JC, Mietzsch M, Singh A, Jimenez Ybargollin A, Kailasan S, Chipman P, Bhattacharya N, Fakhiri J, Grimm D, Kapoor A, Kučinskaitė-Kodzė I, Žvirblienė A, Söderlund-Venermo M, McKenna R, Agbandje-McKenna M. Characterization of the GBoV1 Capsid and Its Antibody Interactions. Viruses. 2021 Feb 20;13(2). doi: 10.3390/v13020330. PubMed PMID: 33672786; PubMed Central PMCID: PMC7924616.

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Bubeck F, Grimm D. 'Hit and run' therapy averts macular degeneration. Nat Biomed Eng. 2021 Feb;5(2):132-133. doi: 10.1038/s41551-021-00690-4. PubMed PMID: 33580229.

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El Andari J, Grimm D. Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors. Biotechnol J. 2021 Jan;16(1):e2000025. doi: 10.1002/biot.202000025. Epub 2020 Oct 7. Review. PubMed PMID: 32975881.

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Senís E, Mosteiro L, Grimm D, Abad M. A Versatile In Vivo System to Study Myc in Cell Reprogramming. Methods Mol Biol. 2021;2318:267-279. doi: 10.1007/978-1-0716-1476-1_14. PubMed PMID: 34019296.

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Weinmann J, Weis S, Sippel J, Tulalamba W, Remes A, El Andari J, Herrmann AK, Pham QH, Borowski C, Hille S, Schönberger T, Frey N, Lenter M, VandenDriessche T, Müller OJ, Chuah MK, Lamla T, Grimm D. Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants. Nat Commun. 2020 Oct 28;11(1):5432. doi: 10.1038/s41467-020-19230-w. PubMed PMID: 33116134; PubMed Central PMCID: PMC7595228.

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Klein S, Müller TG, Khalid D, Sonntag-Buck V, Heuser AM, Glass B, Meurer M, Morales I, Schillak A, Freistaedter A, Ambiel I, Winter SL, Zimmermann L, Naumoska T, Bubeck F, Kirrmaier D, Ullrich S, Barreto Miranda I, Anders S, Grimm D, Schnitzler P, Knop M, Kräusslich HG, Dao Thi VL, Börner K, Chlanda P. SARS-CoV-2 RNA Extraction Using Magnetic Beads for Rapid Large-Scale Testing by RT-qPCR and RT-LAMP. Viruses. 2020 Aug 7;12(8). doi: 10.3390/v12080863. PubMed PMID: 32784757; PubMed Central PMCID: PMC7472728.

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Mathony J, Harteveld Z, Schmelas C, Upmeier Zu Belzen J, Aschenbrenner S, Sun W, Hoffmann MD, Stengl C, Scheck A, Georgeon S, Rosset S, Wang Y, Grimm D, Eils R, Correia BE, Niopek D. Computational design of anti-CRISPR proteins with improved inhibition potency. Nat Chem Biol. 2020 Jul;16(7):725-730. doi: 10.1038/s41589-020-0518-9. Epub 2020 Apr 13. PubMed PMID: 32284602.

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Fakhiri J, Linse KP, Mietzsch M, Xu M, Schneider MA, Meister M, Schildgen O, Schnitzler P, Soderlund-Venermo M, Agbandje-McKenna M, Grimm D. Impact of Natural or Synthetic Singletons in the Capsid of Human Bocavirus 1 on Particle Infectivity and Immunoreactivity. J Virol. 2020 May 18;94(11). doi: 10.1128/JVI.00170-20. Print 2020 May 18. PubMed PMID: 32213611; PubMed Central PMCID: PMC7269442.

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Michler T, Kosinska AD, Festag J, Bunse T, Su J, Ringelhan M, Imhof H, Grimm D, Steiger K, Mogler C, Heikenwalder M, Michel ML, Guzman CA, Milstein S, Sepp-Lorenzino L, Knolle P, Protzer U. Knockdown of Virus Antigen Expression Increases Therapeutic Vaccine Efficacy in High-Titer Hepatitis B Virus Carrier Mice. Gastroenterology. 2020 May;158(6):1762-1775.e9. doi: 10.1053/j.gastro.2020.01.032. Epub 2020 Jan 28. PubMed PMID: 32001321.