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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;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;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

Bak R, Dever DP, Porteus MH. CRISPR/Cas9 genome editing in human hematopoietic stem cells. Nature Protocols (Print). 2018;13(2):358-376. doi: 10.1038/nprot.2017.143

Bak RO, Gomez-Ospina N, Porteus MH. Gene Editing on Center Stage. Trends in Genetics. 2018;34(8):600-611. doi: 10.1016/j.tig.2018.05.004

Bak RO, Dever DP, Porteus MH. Therapeutic Genome Editing in Human Hematopoietic Stem and Progenitor Cells. In Genome Editing and Engineering: From TALENs, ZFNs and CRISPRs to Molecular Surgery. Cambridge University Press. 2018. p. 301-312 doi: 10.1017/9781316756300.022

Charlesworth CT, Camarena J, Cromer MK, Vaidyanathan S, Bak RO, Carte JM et al. Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting. Molecular Therapy - Nucleic Acids. 2018 Sept 7;12:89-104. doi: 10.1016/j.omtn.2018.04.017

Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J et al. Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34 Hematopoietic Stem and Progenitor Cells. Molecular Therapy. 2018;26(10):2431-2442. doi: 10.1016/j.ymthe.2018.06.002

Dalsgaard T , Kaadt CRC , Askou AL , Bak RO , Andersen PO, Hougaard D et al. Improved Lentiviral Gene Delivery to Mouse Liver by Hydrodynamic Vector Injection through Tail Vein. Molecular Therapy - Nucleic Acids. 2018 Sept 7;12:672-683. Epub 2018 Aug 6. doi: 10.1016/j.omtn.2018.07.005

Hollensen AK , Andersen S , Hjorth K , Bak RO , Hansen TB , Kjems J et al. Enhanced Tailored MicroRNA Sponge Activity of RNA Pol II-Transcribed TuD Hairpins Relative to Ectopically Expressed ciRS7-Derived circRNAs. Molecular Therapy - Nucleic Acids. 2018 Dec 7;13:365-375. Epub 2018 Sept 21. doi: 10.1016/j.omtn.2018.09.009

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

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

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

Bak RO, Porteus MH. CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors. Cell Reports. 2017 Jul 18;20(3):750-756. doi: 10.1016/j.celrep.2017.06.064

Bak RO, Dever DP, Reinisch A, Cruz Hernandez D, Majeti R, Porteus MH. Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6. eLife. 2017 Sept 28;6:e27873. doi: 10.7554/eLife.27873

Hollensen AK , Thomsen R , Bak RO , Petersen CC , Ermegaard ER , Aagaard L et al. Improved microRNA suppression by WPRE-linked Tough Decoy microRNA sponges. RNA. 2017 Aug;23(8):1247-1258. Epub 2017 May 9. doi: 10.1261/rna.061192.117

Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE et al. CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134

Displaying results 1 to 50 out of 72

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Revised 07.03.2022

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Rasmus O. Bak