About Aarhus University
Rasmus O. Bak
Associate Professor
Department of Biomedicine - Forskning og uddannelse, Skou-bygningen
Høegh-Guldbergs Gade 10
building 1115, 330
8000 Aarhus C
Denmark
Phone: +4587153562
Mobile: +4593929100
Research areas
Profile
I am a research scientist with a strong background in developing strategies and applying novel techniques for genome engineering in human cells. I have >14 years of research experience during which I have worked with gene therapy in genetic diseases and immuno-oncology. I have experience with a wide panel of gene expression vectors, such as plasmids, transposons, lentiviral vectors, and adeno-associated viral vectors. In addition, I have expertise in several CRISPR/Cas-based platforms such as knockout, knockin, gene replacement, transcriptional regulation, base editing, and prime editing. I have developed chemically modified guide RNAs for use in the CRISPR/Cas9 system and shown that they can facilitate very high levels of genome editing in primary human hematopoietic stem and progenitor cells (Nature Biotechnology, Sept. 2015). I used this platform to develop a potent CRISPR/Cas9-based genome editing strategy for Sickle Cell Disease (Nature, Nov. 2016; Nature Protocols, Jan. 2018). I have also demonstrated that the system supports integration of large transgenes by sequential homologous recombination (Cell Reports, July 2017) and multiplexed genome editing (eLife, Sept. 2017). Finally, I have developed precision tools for manipulating transcriptional of individual genes, aka. CRISPRa and CRISPRi, (Genome Research, 2022), and developed and applied prime editing for liver-directed gene therapy (Molecular Therapy, 2022)
- Published
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
Cromer, M. K., Camarena, J., Martin, R. M., Lesch, B. J., Vakulskas, C. A., Bode, N. M., Kurgan, G., Collingwood, M. A., Rettig, G. R., Behlke, M. A., Lemgart, V. T., Zhang, Y., Goyal, A., Zhao, F., Ponce, E., Srifa, W., Bak, R. O., Uchida, N., Majeti, R., Sheehan, V. A., & 3 othersTisdale, J. F., Dever, D. P. & Porteus, M. H., Apr 2021, In: Nature Medicine. 27, 4, p. 677-687 11 p.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
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STEEP mediates STING ER exit and activation of signaling
Zhang, B. C., Nandakumar, R., Reinert, L. S., Huang, J., Laustsen, A., Gao, Z. L., Sun, C. L., Jensen, S. B., Troldborg, A., Assil, S., Berthelsen, M. F., Scavenius, C., Zhang, Y., Windross, S. J., Olagnier, D., Prabakaran, T., Bodda, C., Narita, R., Cai, Y., Zhang, C. G., & 11 othersStenmark, H., Doucet, C. M., Noda, T., Guo, Z., Goldbach-Mansky, R., Hartmann, R., Chen, Z. J., Enghild, J. J., Bak, R. O., Thomsen, M. K. & Paludan, S. R., 2020, In: Nature Immunology. 21, 8, p. 868-879 12 p.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
Vakulskas, C. A., Dever, D. P., Rettig, G. R., Turk, R., Jacobi, A. M., Collingwood, M. A., Bode, N. M., McNeill, M. S., Yan, S., Camarena, J., Lee, C. M., Park, S. H., Wiebking, V., Bak, R. O., Gomez-Ospina, N., Pavel-Dinu, M., Sun, W., Bao, G., Porteus, M. H. & Behlke, M. A., 2018, In: Nature Medicine. 24, 8, p. 1216-1224 9 p.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
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CRISPR/Cas9 genome editing in human hematopoietic stem cells
Bak, R., Dever, D. P. & Porteus, M. H., 2018, In: Nature Protocols (Print). 13, 2, p. 358-376 19 p.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
Dever, D. P., Bak, R. O., Reinisch, A., Camarena, J., Washington, G., Nicolas, C. E., Pavel-Dinu, M., Saxena, N., Wilkens, A. B., Mantri, S., Uchida, N., Hendel, A., Narla, A., Majeti, R., Weinberg, K. I. & Porteus, M. H., 17 Nov 2016, In: Nature. 539, 7629, p. 384-389 6 p.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
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Activation of proto-oncogenes by disruption of chromosome neighborhoods
Hnisz, D., Weintraub, A. S., Day, D. S., Valton, A-L., Bak, R. O., Li, C. H., Goldmann, J., Lajoie, B. R., Fan, Z. P., Sigova, A. A., Reddy, J., Borges-Rivera, D., Lee, T. I., Jaenisch, R., Porteus, M. H., Dekker, J. & Young, R. A., 25 Mar 2016, In: Science. 351, 6280, p. 1454-1458 5 p.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
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Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
Hendel, A., Bak, R. O., Clark, J. T., Kennedy, A. B., Ryan, D. E., Roy, S., Steinfeld, I., Lunstad, B. D., Kaiser, R. J., Wilkens, A. B., Bacchetta, R., Tsalenko, A., Dellinger, D., Bruhn, L. & Porteus, M. H., 29 Jun 2015, In: Nature Biotechnology.Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Journal article › Research › peer-review
CRISPR – DNA-saksen der bekæmper arvelige sygdomme
Activity: Talk or presentation types › Lecture and oral contribution
CRISPR – DNA-saksen der bekæmper arvelige sygdomme
Activity: Talk or presentation types › Lecture and oral contribution
Precise Genome Editing of Hematopoietic Stem Cells
Activity: Talk or presentation types › Lecture and oral contribution
AIAS Fellow Rasmus O. Bak and collaborators receive large grant from Innovation Fund Denmark
Press/Media: Press / Media
Biomedicine provides three new Lundbeck Foundation Fellows
Press/Media: Press / Media
AIAS Fellow Rasmus O. Bak new Lundbeck Foundation Fellow
Press/Media: Press / Media
ID: 14965020