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In vivo CRISPR editing with no detectable genome-wide off-target mutations.
Akcakaya P, Bobbin ML, Guo JA, Malagon-Lopez J, Clement K, Garcia SP, Fellows MD, Porritt MJ, Firth MA, Carreras A, Baccega T, Seeliger F, Bjursell M, Tsai SQ, Nguyen NT, Nitsch R, Mayr LM, Pinello L, Bohlooly-Y M, Aryee MJ, Maresca M, Joung JK. Akcakaya P, et al. Among authors: firth ma. Nature. 2018 Sep;561(7723):416-419. doi: 10.1038/s41586-018-0500-9. Epub 2018 Sep 12. Nature. 2018. PMID: 30209390 Free PMC article.
Universal toxin-based selection for precise genome engineering in human cells.
Li S, Akrap N, Cerboni S, Porritt MJ, Wimberger S, Lundin A, Möller C, Firth M, Gordon E, Lazovic B, Sieńska A, Pane LS, Coelho MA, Ciotta G, Pellegrini G, Sini M, Xu X, Mitra S, Bohlooly-Y M, Taylor BJM, Sienski G, Maresca M. Li S, et al. Among authors: firth m. Nat Commun. 2021 Jan 21;12(1):497. doi: 10.1038/s41467-020-20810-z. Nat Commun. 2021. PMID: 33479216 Free PMC article.
Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes.
Zabulica M, Srinivasan RC, Akcakaya P, Allegri G, Bestas B, Firth M, Hammarstedt C, Jakobsson T, Jakobsson T, Ellis E, Jorns C, Makris G, Scherer T, Rimann N, van Zuydam NR, Gramignoli R, Forslöw A, Engberg S, Maresca M, Rooyackers O, Thöny B, Häberle J, Rosen B, Strom SC. Zabulica M, et al. Mol Ther. 2021 May 5;29(5):1903-1917. doi: 10.1016/j.ymthe.2021.01.024. Epub 2021 Jan 21. Mol Ther. 2021. PMID: 33484963 Free PMC article.
Author Correction: Universal toxin-based selection for precise genome engineering in human cells.
Li S, Akrap N, Cerboni S, Porritt MJ, Wimberger S, Lundin A, Möller C, Firth M, Gordon E, Lazovic B, Sieńska A, Pane LS, Coelho MA, Ciotta G, Pellegrini G, Sini M, Xu X, Mitra S, Bohlooly-Y M, Taylor BJM, Sienski G, Maresca M. Li S, et al. Among authors: firth m. Nat Commun. 2021 May 10;12(1):2832. doi: 10.1038/s41467-021-23345-z. Nat Commun. 2021. PMID: 33972550 Free PMC article. No abstract available.
Rapid target validation in a Cas9-inducible hiPSC derived kidney model.
Shamshirgaran Y, Jonebring A, Svensson A, Leefa I, Bohlooly-Y M, Firth M, Woollard KJ, Hofherr A, Rogers IM, Hicks R. Shamshirgaran Y, et al. Sci Rep. 2021 Aug 16;11(1):16532. doi: 10.1038/s41598-021-95986-5. Sci Rep. 2021. PMID: 34400685 Free PMC article.
Engineered Cas9 extracellular vesicles as a novel gene editing tool.
Osteikoetxea X, Silva A, Lázaro-Ibáñez E, Salmond N, Shatnyeva O, Stein J, Schick J, Wren S, Lindgren J, Firth M, Madsen A, Mayr LM, Overman R, Davies R, Dekker N. Osteikoetxea X, et al. J Extracell Vesicles. 2022 May;11(5):e12225. doi: 10.1002/jev2.12225. J Extracell Vesicles. 2022. PMID: 35585651 Free PMC article.
Simultaneous inhibition of DNA-PK and Polϴ improves integration efficiency and precision of genome editing.
Wimberger S, Akrap N, Firth M, Brengdahl J, Engberg S, Schwinn MK, Slater MR, Lundin A, Hsieh PP, Li S, Cerboni S, Sumner J, Bestas B, Schiffthaler B, Magnusson B, Di Castro S, Iyer P, Bohlooly-Y M, Machleidt T, Rees S, Engkvist O, Norris T, Cadogan EB, Forment JV, Šviković S, Akcakaya P, Taheri-Ghahfarokhi A, Maresca M. Wimberger S, et al. Nat Commun. 2023 Aug 14;14(1):4761. doi: 10.1038/s41467-023-40344-4. Nat Commun. 2023. PMID: 37580318 Free PMC article.
50 results