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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 CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA. Vakulskas CA, et al. Among authors: bak ro. Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6. Nat Med. 2018. PMID: 30082871 Free PMC article.
Impact of U2AF1 mutations on circular RNA expression in myelodysplastic neoplasms.
Wedge E, Ahmadov U, Hansen TB, Gao Z, Tulstrup M, Côme C, Nonavinkere Srivatsan S, Ahmed T, Jespersen JS, Schlotmann BC, Schöllkopf C, Raaschou-Jensen K, Ødum N, Kjems J, Bak RO, Walter MJ, Grønbæk K, Kristensen LS. Wedge E, et al. Among authors: bak ro. Leukemia. 2023 May;37(5):1113-1125. doi: 10.1038/s41375-023-01866-4. Epub 2023 Mar 15. Leukemia. 2023. PMID: 36922625
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.
Hendel A, Bak RO, Clark JT, Kennedy AB, Ryan DE, Roy S, Steinfeld I, Lunstad BD, Kaiser RJ, Wilkens AB, Bacchetta R, Tsalenko A, Dellinger D, Bruhn L, Porteus MH. Hendel A, et al. Among authors: bak ro. Nat Biotechnol. 2015 Sep;33(9):985-989. doi: 10.1038/nbt.3290. Epub 2015 Jun 29. Nat Biotechnol. 2015. PMID: 26121415 Free PMC article.
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. Dever DP, et al. Among authors: bak ro. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7. Nature. 2016. PMID: 27820943 Free PMC article.
Gene Editing on Center Stage.
Bak RO, Gomez-Ospina N, Porteus MH. Bak RO, et al. Trends Genet. 2018 Aug;34(8):600-611. doi: 10.1016/j.tig.2018.05.004. Epub 2018 Jun 13. Trends Genet. 2018. PMID: 29908711 Review.
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.
Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J, Kaushik M, Hay SR, Martin RM, Steinfeld I, Bak RO, Dever DP, Hendel A, Bruhn L, Porteus MH. Cromer MK, et al. Among authors: bak ro. Mol Ther. 2018 Oct 3;26(10):2431-2442. doi: 10.1016/j.ymthe.2018.06.002. Epub 2018 Jul 11. Mol Ther. 2018. PMID: 30005866 Free PMC article.
72 results