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Leukemia|July 18, 2023
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 vivoSigne Neldeborg, Johannes Frasez Soerensen, Charlotte Thornild Møller, et al.
Nature Communications|March 7, 2024
CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expressionHuiqiang Cai, Bin Zhang, Johanne Ahrenfeldt, et al.
Nature Communications|September 1, 2018
Interferon priming is essential for human CD34+ cell-derived plasmacytoid dendritic cell maturation and functionA Laustsen, R O Bak, C Krapp, et al.
Nature Biotechnology|June 30, 2015
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cellsAyal Hendel, Rasmus O Bak, Joseph T Clark, et al.
Nucleic Acids Research|August 14, 2025
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 nanoparticlesSofie Andersen, Jonas Holst Wolff, Thomas Wisbech Skov, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 15, 2018
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34<sup>+</sup> Hematopoietic Stem and Progenitor CellsM Kyle Cromer, Sriram Vaidyanathan, Daniel E Ryan, et al.
Nature|November 8, 2016
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cellsDaniel P Dever, Rasmus O Bak, Andreas Reinisch, et al.
Proceedings of the National Academy of Sciences of the United States of America|October 25, 2013
IFI16 senses DNA forms of the lentiviral replication cycle and controls HIV-1 replicationMartin R Jakobsen, Rasmus O Bak, Annika Andersen, et al.
Nature Communications|August 12, 2025
Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous diseaseJonas Holst Wolff, Thomas Wisbech Skov, Didde Haslund, et al.
Nature Communications|September 8, 2019
Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type INatalia Gomez-Ospina, Samantha G Scharenberg, Nathalie Mostrel, et al.
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