Bak Lab

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Andersen S, Wolff JH, Skov TW, Janns JH, Davis LJ, Haldrup JH et al. Gene editing in hematopoietic stem cells by co-delivery of Cas9/sgRNA ribonucleoprotein and templates for homology-directed repair in 'all-in-one' lentivirus-derived nanoparticles. Nucleic Acids Research. 2025 Aug 28;53(15):gkaf767. doi: 10.1093/nar/gkaf767
Bak RO, Holm M, Møller B, Mikkelsen JG, Mogensen TH. CRISPR/Cas-geneditering af humane stamceller til kurativ behandling af primær immundefekt. Ugeskrift for Læger. 2025 May 26;187(22):1-7. doi: 10.61409/V02250083
Broksø AD, Bendixen L, Fammé S, Mikkelsen K, Jensen TI, Bak RO. Orthogonal transcriptional modulation and gene editing using multiple CRISPR/Cas systems. Molecular Therapy. 2025 Jan 8;33(1):71-89. Epub 2024 Nov 19. doi: 10.1016/j.ymthe.2024.11.024
Dahl-Jessen M, Terkelsen T, Bak RO, Jensen UB. Characterization of the role of spatial proximity of DNA double-strand breaks in the formation of CRISPR-Cas9-induced large structural variations. Genome Research. 2025;35(2):231-241. Epub 2025 Jan 13. doi: 10.1101/gr.278575.123
Gao Z, Bak RO. Integration of large genetic payloads using prime editing and site-specific integrases. Nature Protocols. 2025 Jun;20(6):1391-1392. Epub 2024 Dec 15. doi: 10.1038/s41596-024-01094-9
Hinke DM, Dorset SR, Bratland E, Wolff JH, Olsnes AM, Mikkelsen JG et al. CXCR2 deficiency with myelokathexis caused by a novel variant: correction via CRISPR/Cas9. Haematologica. 2025 Aug 28. Epub 2025 Aug 28. doi: 10.3324/haematol.2025.288111
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 Aug 26. Epub 2025 Aug 26. doi: 10.1016/j.ymthe.2025.08.032
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
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
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 Jul 29. Epub 2025 Jul 29. doi: 10.1016/j.ymthe.2025.07.038
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
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
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 Sept 19. Epub 2025 Sept 19. doi: 10.1038/s41590-025-02283-8
Cai H, Zhang B, Ahrenfeldt J, Joseph JV, Riedel M, Gao Z et al. CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expression. Nature Communications. 2024 Dec;15(1):2088. doi: 10.1038/s41467-024-46370-0, 10.1038/s41467-024-46370-0
Chai C, Liang L, Mikkelsen NS, Wang W, Zhao W, Sun C et al. Single-cell transcriptome analysis of epithelial, immune, and stromal signatures and interactions in human ovarian cancer. Communications Biology. 2024 Jan 26;7(1):131. 131. doi: 10.1038/s42003-024-05826-1
Dudek AM, Feist WN, Sasu EJ, Luna SE, Ben-Efraim K, Bak RO et al. A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection. Cell Stem Cell. 2024 Apr;31(4):499-518.e6. doi: 10.1016/j.stem.2024.03.002
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
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
Bendixen L, Jensen TI, Bak RO. CRISPR-Cas-mediated transcriptional modulation: The therapeutic promises of CRISPRa and CRISPRi. Molecular Therapy. 2023 Jul;31(7):1920-1937. Epub 2023 Mar 23. doi: 10.1016/j.ymthe.2023.03.024
Cavazza A, Hendel A, Bak RO, Rio P, Güell M, Lainšček D et al. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi. Molecular Therapy Nucleic Acids. 2023 Dec 12;34:102066. 102066. doi: 10.1016/j.omtn.2023.102066, 10.1016/j.omtn.2023.102066
Dorset SR, Bak RO. The p53 challenge of hematopoietic stem cell gene editing. Molecular Therapy Methods and Clinical Development. 2023 Sept;30:83-89. doi: 10.1016/j.omtm.2023.06.003
Galdos FX, Lee C, Lee S, Paige S, Goodyer W, Xu S et al. Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation. eLife. 2023 Jun 7;12:e80075. doi: 10.7554/eLife.80075
He M, Roussak K, Ma F, Borcherding N, Garin V, White M et al. CD5 expression by dendritic cells directs T cell immunity and sustains immunotherapy responses. Science. 2023 Feb 17;379(6633):eabg2752. doi: 10.1126/science.abg2752
Hendel A, Bak RO. Editorial: CRISPR and beyond: Cutting-edge technologies for gene correction in therapeutic applications. Frontiers in Genome Editing. 2023;5:1203864. doi: 10.3389/fgeed.2023.1203864
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
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
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. The EMBO Journal. 2023 Oct;42(19):e113118. Epub 2023 Aug 30. doi: 10.15252/embj.2022113118
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
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
Gao Z, Ravendran S, Mikkelsen NS, Haldrup J, Cai H, Ding X et al. A truncated reverse transcriptase enhances prime editing by split AAV vectors. Molecular Therapy. 2022 Sept;30(9):2942-2951. doi: 10.1016/j.ymthe.2022.07.001
Hernández SS, Jakobsen MR, Bak RO. Plasmacytoid Dendritic Cells as a Novel Cell-Based Cancer Immunotherapy. International Journal of Molecular Sciences . 2022 Sept;23(19):11397. doi: 10.3390/ijms231911397
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
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
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
Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM et al. Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells. Nature Medicine. 2021 Apr;27(4):677-687. Epub 2021 Mar 18. doi: 10.1038/s41591-021-01284-y
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
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
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
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
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
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, 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
Axelgaard E, Jensen TI, Bak RO. Therapeutic gene editing in haematological disorders with CRISPR/Cas9. British Journal of Haematology. 2019;185(5):821-835. Epub 2019 Mar 13. doi: 10.1111/bjh.15851
Bak RO. The Potential of CRISPR/Cas9 in Hematotherapy. Stem Cells and Development. 2019;28(11):710-711. Epub 2019 May 15. doi: 10.1089/scd.2019.0079
Dever DP, Scharenberg SG, Camarena J, Kildebeck EJ, Clark JT, Martin RM et al. CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells. iScience. 2019 May 31;15:524-535. doi: 10.1016/j.isci.2019.04.036
Gomez-Ospina N, Scharenberg SG, Mostrel N, Bak RO, Mantri S, Quadros RM et al. Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I. Nature Communications. 2019 Dec 1;10(1):4045. 4045. doi: 10.1038/s41467-019-11962-8
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
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

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Revised 11.09.2025
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Rasmus O. Bak