Billede af Rasmus Bak Photo by Simon Byrial Fischel, Aarhus University
  • Høegh-Guldbergs Gade 10, 1115, 330

    8000 Aarhus C

    Danmark

20072024

Publikationer pr. år

Personlig profil

Profil

Profil
I am a research scientist with a strong background in developing strategies and applying novel techniques for genome engineering in human cells. I have >16 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)

Emneord

  • Genterapi
  • CRISPR
  • Genmedicin
  • Genredigering
  • Genetik
  • Genetisk behandling
  • CRISPR/Cas9

Fagområder

  • CRISPR/Cas
  • Geneditering
  • Genome Engineering
  • Celleterapi

Fingeraftryk

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Samarbejde og topforskningsområder i de sidste fem år

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  • 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 flere, Tisdale, J. F., Dever, D. P. & Porteus, M. H., apr. 2021, I: Nature Medicine. 27, 4, s. 677-687 11 s.

    Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

    Åben adgang
    52 Citationer (Scopus)
  • 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, I: Nature. 539, 7629, s. 384-389 6 s.

    Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

    659 Citationer (Scopus)
  • 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, I: Science. 351, 6280, s. 1454-1458 5 s.

    Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

    717 Citationer (Scopus)
  • 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, I: Nature Biotechnology.

    Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

    812 Citationer (Scopus)
  • CD5 expression by dendritic cells directs T cell immunity and sustains immunotherapy responses

    He, M., Roussak, K., Ma, F., Borcherding, N., Garin, V., White, M., Schutt, C., Jensen, T. I., Zhao, Y., Iberg, C. A., Shah, K., Bhatia, H., Korenfeld, D., Dinkel, S., Gray, J., Ulezko Antonova, A., Ferris, S., Donermeyer, D., Lindestam Arlehamn, C. & Gubin, M. M. & 11 flere, Luo, J., Gorvel, L., Pellegrini, M., Sette, A., Tung, T., Bak, R., Modlin, R. L., Fields, R. C., Schreiber, R. D., Allen, P. M. & Klechevsky, E., feb. 2023, I: Science. 379, 6633, 17 s., eabg2752.

    Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

    36 Citationer (Scopus)