Bak Lab

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Rasmus O. Bak on Google Scholar profile

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Zhang BC, Nandakumar R, Reinert LS, Huang J, Laustsen A, Gao ZL et al. STEEP mediates STING ER exit and activation of signaling. Nature Immunology. 2020;21(8):868-879. doi: 10.1038/s41590-020-0730-5
Zhang B, Nandakumar R, Reinert LS, Huang J, Laustsen A, Gao Z et al. Author Correction: STEEP mediates STING ER exit and activation of signaling (Nature Immunology, (2020), 21, 8, (868-879), 10.1038/s41590-020-0730-5). Nature Immunology. 2020 Nov;21(11):1468-1469. doi: 10.1038/s41590-020-0803-5
Zhang BC, Pedersen A, Reinert LS, Li Y, Narita R, Idorn M et al. STING signals to NF-κB from late endolysosomal compartments using IRF3 as an adaptor. Nature Immunology. 2025 Nov;26(11):1916-1930. Epub 2025 Sept 19. doi: 10.1038/s41590-025-02283-8
Wolff JH, Skov TW, Haslund D, Dorset SR, Revenfeld ALS, Aussel C et al. Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease. Nature Communications. 2025 Dec;16(1):7475. doi: 10.1038/s41467-025-62738-2
Wiebking V, Lee CM, Mostrel N, Lahiri P, Bak R, Bao G et al. Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell transplantation. Haematologica. 2021 Mar;106(3):847-858. doi: 10.3324/haematol.2019.233882
Wedge E, Ahmadov U, Hansen TB, Gao Z, Tulstrup M, Côme C et al. Impact of U2AF1 mutations on circular RNA expression in myelodysplastic neoplasms. Leukemia. 2023 May;37(5):1113-1125. Epub 2023 Mar 15. doi: 10.1038/s41375-023-01866-4
van der Sluis RM, Cham LB, Gris-Oliver A, Gammelgaard KR, Pedersen JG, Idorn M et al. TLR2 and TLR7 mediate distinct immunopathological and antiviral plasmacytoid dendritic cell responses to SARS-CoV-2 infection. EMBO Journal. 2022 May;41(10):e109622. doi: 10.15252/embj.2021109622
van der Sluis RM, García-Rodríguez JL, Nielsen IH, Gris-Oliver A, Becker J, Costa B et al. Distinctive CD8+ T cell activation by antigen-presenting plasmacytoid dendritic cells compared to conventional dendritic cells. Cell Reports. 2025 Mar 25;44(3):115413. doi: 10.1016/j.celrep.2025.115413
van der Horst D, Kurmasheva N, Marqvorsen MHS, Assil S, Rahimic AHF, Kollmann CF et al. SAM68 directs STING signaling to apoptosis in macrophages. Communications Biology. 2024 Mar;7:283. 283. doi: 10.1038/s42003-024-05969-1
Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA et al. A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells. Nature Medicine. 2018;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0
Terkelsen T, Mikkelsen NS, Bak EN, Vad-Nielsen J, Blechingberg J, Weiss S et al. CRISPR activation to characterize splice-altering variants in easily accessible cells. American Journal of Human Genetics. 2024 Feb 1;111(2):309-322. doi: 10.1016/j.ajhg.2023.12.024
Terkelsen T, Yumiceba V, Kim J, Melo US, Axelgaard E, Febbraro F et al. Position effect at the SOX3 locus by an interchromosomal insertion causes hereditary spastic paraplegia. American Journal of Human Genetics. 2026 Aug 6;113(8):1736-1753. Epub 2026 Jul 24. doi: 10.1016/j.ajhg.2026.07.001
Staunstrup NH, Sharma N, Bak RO, Svensson L, Petersen TK, Aarenstrup L et al. A Sleeping Beauty DNA transposon-based genetic sensor for functional screening of vitamin D3 analogues. BMC Biotechnology. 2011 Apr 7;11(1):33. doi: 10.1186/1472-6750-11-33
Staunstrup NH, Bak RO, Cai Y, Svensson L, Petersen AT, Rosada C et al. A lentiviral vector-based genetic sensor system for comparative analysis of permeability and activity of vitamin D3 analogues in xenotransplanted human skin. Experimental Dermatology. 2013 Mar 22;22(3):178-83. doi: 10.1111/exd.12091
Skov TW, Wolff JH, Haslund D, Revenfeld AL, van de Venn L, Dorset SR et al. Treatment of GATA2 deficiency by allele-specific CRISPR-Cas9-directed gene correction in hematopoietic stem cells. Molecular Therapy. 2025 Nov 5;33(11):5644-5660. Epub 2025 Jul 29. doi: 10.1016/j.ymthe.2025.07.038
Skoczek D, Hohendorff J, Malecki MT, Roig-Merino A, Bak RO, Kachamakova-Trojanowska N. CRISPR/Cas9-based repair of a heterozygous HNF1A mutation in patient-derived hiPSCs. Human Genetics. 2026 Dec;145(1):69. doi: 10.1007/s00439-026-02863-0
Skipper KA, Nielsen MG, Andersen S, Ryø LB, Bak RO, Mikkelsen JG. Time-Restricted PiggyBac DNA Transposition by Transposase Protein Delivery Using Lentivirus-Derived Nanoparticles. Molecular Therapy - Nucleic Acids. 2018 Jun 1;11:253-262. doi: 10.1016/j.omtn.2018.02.006
Sharma N, Hollensen AK, Bak RO, Staunstrup NH, Schrøder LD, Mikkelsen JG. The Impact of cHS4 Insulators on DNA Transposon Vector Mobilization and Silencing in Retinal Pigment Epithelium Cells. PLoS One. 2012;7(10). doi: 10.1371/journal.pone.0048421
Sharma N, Cai Y, Bak RO, Jakobsen MR, Schrøder LD, Mikkelsen JG. Efficient sleeping beauty DNA transposition from DNA minicircles. Molecular Therapy - Nucleic Acids. 2013;2:e74. doi: 10.1038/mtna.2013.1
Sánchez Hernández S, Bjerg TW, Nielsen IH, Laustsen A, Q Tang H, Pedersen LH et al. Characterization of TLR9 responsiveness in cell subsets derived from in vitro pDC differentiation of hematopoietic stem and progenitor cells. Frontiers in Immunology. 2025;16:1550397. doi: 10.3389/fimmu.2025.1550397
Sánchez Hernández S, Bjerg TW, Helstrup Nielsen I, Laustsen A, Q Tang H, Henning Pedersen L et al. Corrigendum: Characterization of TLR9 responsiveness in cell subsets derived from in vitro pDC differentiation of hematopoietic stem and progenitor cells. Frontiers in Immunology. 2025;16:1625107. doi: 10.3389/fimmu.2025.1625107
Şahin C, Formica TM, Silva A, Della Pelle G, Davis MC, Andersen DG et al. The landscape and trajectory of global CRISPR therapeutics. Molecular Therapy Nucleic Acids. 2026 Sept 8;37(3):103011. doi: 10.1016/j.omtn.2026.103011
Rothemejer FH, Lauritsen NP, Juhl AK, Schleimann MH, König S, Søgaard OS et al. Development of HIV-Resistant CAR T Cells by CRISPR/Cas-Mediated CAR Integration into the CCR5 Locus. Viruses. 2023 Jan;15(1):202. doi: 10.3390/v15010202
Ren F, Narita R, Rashidi AS, Fruhwürth S, Gao Z, Bak RO et al. ER stress induces caspase-2-tBID-GSDME-dependent cell death in neurons lytically infected with herpes simplex virus type 2. EMBO Journal. 2023 Oct;42(19):e113118. Epub 2023 Aug 30. doi: 10.15252/embj.2022113118
Ravendran S, Hernández SS, König S, Bak RO. CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome. Frontiers in Genome Editing. 2022 Mar;4:793010. doi: 10.3389/fgeed.2022.793010
Ravendran S, Fammé S, Noer MG, Skov TW, Mikkelsen NS, Lee Schneller J et al. AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality. Molecular therapy. Advances. 2026 Sept 10;34(3):201787. doi: 10.1016/j.omta.2026.201787
Rasmussen TK, Andersen T, Bak RO, Yiu G, Sørensen C, Stengaard-Pedersen K et al. Overexpression of MicroRNA-155 increases IL-21 mediated STAT3 signaling and IL-21 production in systemic lupus erythematosus. Arthritis research & therapy. 2015 Jun 9;17(1):154. doi: 10.1186/s13075-015-0660-z
Qiu L, Ungerbäck JT, Bennett EP, Bak RO. Biologic Therapies for Alleviating Neurodegeneration in Lysosomal Storage Diseases. BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy. 2026 Sept 14. Epub 2026 Sept 14. doi: 10.1007/s40259-026-00804-x
Pedersen MG, Møller BK, Bak RO. Recent Advances in the Development of Anti-FLT3 CAR T-Cell Therapies for Treatment of AML. Biomedicines. 2022 Oct;10(10):2441. doi: 10.3390/biomedicines10102441
Nitulescu AM, Du W, Glaser V, Kath J, Aird EJ, Cullot G et al. Single-stranded HDR templates with truncated Cas12a-binding sequences improve knock-in efficiencies in primary human T cells. Molecular Therapy Nucleic Acids. 2025 Jun;36(2):102568. doi: 10.1016/j.omtn.2025.102568
Neldeborg S, Soerensen JF, Møller CT, Bill M, Gao Z, Bak RO et al. Dual intron-targeted CRISPR-Cas9-mediated disruption of the AML RUNX1-RUNX1T1 fusion gene effectively inhibits proliferation and decreases tumor volume in vitro and in vivo. Leukemia. 2023 Sept;37(9):1792-1801. doi: 10.1038/s41375-023-01950-9
Nascimento Primo M, Bak RO, Schibler B, Mikkelsen JG. Regulation of pro-inflammatory cytokines TNFα and IL24 by microRNA-203 in primary keratinocytes. Cytokine. 2012. doi: 10.1016/j.cyto.2012.07.031
Nascimento Lopes Primo M, Bak RO, Mikkelsen JG. Lentiviral vectors for cutaneous RNA managing. Experimental Dermatology. 2012;21(3):162-70. doi: 10.1111/j.1600-0625.2011.01436.x
Moldt B, Miskey C, Staunstrup NH, Gogol-Döring A, Bak RO, Sharma N et al. Comparative genomic integration profiling of Sleeping Beauty transposons mobilized with high efficacy from integrase-defective lentiviral vectors in primary human cells. Molecular Therapy. 2011 Aug;19(8).
Mikkelsen NS, Hernandez SS, Jensen TI, Schneller JL, Bak RO. Enrichment of transgene integrations by transient CRISPR activation of a silent reporter gene. Molecular Therapy Methods and Clinical Development. 2023 Jun;29:1-16. doi: 10.1016/j.omtm.2023.02.010
Mikkelsen NS, Bak RO. Enrichment strategies to enhance genome editing. Journal of Biomedical Science. 2023 Dec;30(1):51. doi: 10.1186/s12929-023-00943-1
Mikkelsen NS, Ravendran S, Broksø AD, Skjelbostad SF, Pedersen MG, Fang H et al. Orthogonal CRISPR systems for targeted integration and multiplex base editing enable nonviral engineering of allogeneic CAR T cells. Molecular Therapy. 2025 Dec 3;33(12):6082-6100. Epub 2025 Aug 26. doi: 10.1016/j.ymthe.2025.08.032
Mieritz IK, Laustsen C, Axelgaard E, Dorset SR, Bak RO, Bertelsen LB. Glycolytic alterations as biomarkers in polycystic kidney disease: A study using a PKD1 knockout model in NRK-52E rat kidney epithelial cells. Physiological Reports. 2026 Mar;14(6):e70816. doi: 10.14814/phy2.70816
Martin RM, Ikeda K, Cromer MK, Uchida N, Nishimura T, Romano R et al. Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination. Cell Stem Cell. 2019 May 2;24(5):821-828.e5. doi: 10.1016/j.stem.2019.04.001
Lisle TL, Jacobsen EB, Ahmad SM, Met Ö, Bak RO, Andersen MH. Engineering T cells with a novel PD-L1-specific TCR targeting immune and cancer cells. Signal Transduction and Targeted Therapy. 2026 Dec;11(1):246. doi: 10.1038/s41392-026-02798-y
Laustsen A, Bak R, Krapp C, Kjær L, Egedahl JH, Petersen CC et al. Interferon priming is essential for human CD34+ cell-derived plasmacytoid dendritic cell maturation and function. Nature Communications. 2018 Aug 30;9(1):3525. doi: 10.1038/s41467-018-05816-y
Laustsen A, Bak RO. Electroporation-Based CRISPR/Cas9 Gene Editing Using Cas9 Protein and Chemically Modified sgRNAs. In Luo Y, editor, Methods in Molecular Biology: Methods and protocols. New York: Springer Science+Business Media. 2019. p. 127-134. (Methods in Molecular Biology, Vol. 1961). doi: 10.1007/978-1-4939-9170-9_9
Laustsen A, van der Sluis RM, Gris Oliver A, Sanchez Hernandez S, Cemalovic E, Tang HQ et al. Ascorbic acid supports ex vivo generation of plasmacytoid dendritic cells from circulating hematopoietic stem cells. eLife. 2021 Sept;10:e65528. doi: 10.7554/eLife.65528
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA et al. Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease. Science Translational Medicine. 2021 Jun;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444
Kofod-Olsen E, Møller JM, Schleimann MH, Bundgaard B, Bak RO, Øster B et al. Inhibition of p53-dependent, but not p53-independent, cell death by U19 protein from human herpesvirus 6B. PLoS One. 2013;8(e59223):1-11.
Jensen TI, Mikkelsen NS, Gao Z, Foßelteder J, Pabst G, Axelgaard E et al. Targeted regulation of transcription in primary cells using CRISPRa and CRISPRi. Genome Research. 2021 Nov;31(11):2120-2130. Epub 2021 Aug 18. doi: 10.1101/gr.275607.121
Jakobsen MR, Bak RO, Andersen A, Berg RK, Jensen SB, Tengchuan J et al. IFI16 senses DNA forms of the lentiviral replication cycle and controls HIV-1 replication. Proceedings of the National Academy of Sciences (PNAS). 2013 Oct 23. doi: 10.1073/pnas.1311669110
Holmgaard AB, Askou AL, Jensen EG, Alsing S, Bak RO, Mikkelsen JG et al. Targeted Knockout of the Vegfa Gene in the Retina by Subretinal Injection of RNP Complexes Containing Cas9 Protein and Modified sgRNAs. Molecular Therapy. 2021 Jan;29(1):191-207. Epub 2020 Sept 23. doi: 10.1016/j.ymthe.2020.09.032
Holm CK, Rahbek SH, Gad HH, Bak RO, Jakobsen MR, Jiang Z et al. Influenza A virus targets a cGAS-independent STING pathway that controls enveloped RNA viruses. Nature Communications. 2016 Feb 19;7:10680. doi: 10.1038/ncomms10680
Hollensen AK, Bak RO, Haslund D, Mikkelsen JG. Suppression of microRNAs by dual-targeting and clustered Tough Decoy inhibitors. RNA Biology. 2013;10(3).

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Revised 26.03.2026
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Rasmus O. Bak