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CRISPR/Cas9
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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
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
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
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
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
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
Bak RO
, Stenderup K
, Rosada C, Petersen LB, Moldt B, Dagnæs-Hansen F et al.
Targeting of human interleukin-12B by small hairpin RNAs in xenografted psoriatic skin
.
B M C Dermatology
. 2011 Feb 27;11(1):5. doi: 10.1186/1471-5945-11-5
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
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
Cai Y
, Bak R
, Mikkelsen JG
.
Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases
.
eLife
. 2014 Apr 24;3:e01911. doi: 10.7554/eLife.01911
Hollensen AK
, Bak RO
, Haslund D
, Mikkelsen JG
.
Suppression of microRNAs by dual-targeting and clustered Tough Decoy inhibitors
.
RNA Biology
. 2013;10(3).
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
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
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;7(1):283. 283. doi: 10.1038/s42003-024-05969-1
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
Bak RO
, Mikkelsen JG
.
Regulation of cytokines by small RNAs during skin inflammation
.
Journal of Biomedical Science
. 2010;17(53).
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
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;34:102066. doi: 10.1016/j.omtn.2023.102066
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
Bak RO
, Hollensen AK
, Primo MN, Sørensen CD
, Mikkelsen JG
.
Potent microRNA suppression by RNA Pol II-transcribed 'Tough Decoy' inhibitors
.
RNA
. 2013;19(2):280-293. doi: 10.1261/rna.034850.112
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
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
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
Bak RO
, Mikkelsen JG
.
Mobilization of DNA transposable elements from lentiviral vectors
.
Mobile Genetic Elements
. 2011 Jul;1(2):139-144.
Bak R
, Mikkelsen JG
.
miRNA sponges: soaking up miRNAs for regulation of gene expression
.
Wiley Interdisciplinary Reviews: RNA
. 2013 Dec 23. doi: 10.1002/wrna.1213
Bak RO
, Hollensen AK
, Mikkelsen JG
.
Managing MicroRNAs with Vector-Encoded Decoy-Type Inhibitors
.
Molecular Therapy
. 2013 Aug;21(8):1478-85. doi: 10.1038/mt.2013.113
Nascimento Lopes Primo M
, Bak RO
, Mikkelsen JG
.
Lentiviral vectors for cutaneous RNA managing
.
Experimental Dermatology Online
. 2012;21(3):162-70. doi: 10.1111/j.1600-0625.2011.01436.x
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
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
.
P L o S One
. 2013;8(e59223):1-11.
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
, 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
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
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
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
. 2013 Oct 23. doi: 10.1073/pnas.1311669110
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
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
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
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
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
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
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
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
, 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
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 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
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
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
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
Cai Y
, Bak RO
, Krogh LB
, Staunstrup NH
, Moldt B
, Corydon TJ
et al.
DNA transposition by protein transduction of the piggyBac transposase from lentiviral Gag precursors
.
Nucleic Acids Research
. 2013 Nov 21. doi: 10.1093/nar/gkt1163
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Rasmus O. Bak