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Page 1
An aptamer-mediated base editing platform for simultaneous knockin and multiple gene knockout for allogeneic CAR-T cells generation.
Porreca I, Blassberg R, Harbottle J, Joubert B, Mielczarek O, Stombaugh J, Hemphill K, Sumner J, Pazeraitis D, Touza JL, Francescatto M, Firth M, Selmi T, Collantes JC, Strezoska Z, Taylor B, Jin S, Wiggins CM, van Brabant Smith A, Lambourne JJ. Porreca I, et al. Among authors: firth m. Mol Ther. 2024 Aug 7;32(8):2692-2710. doi: 10.1016/j.ymthe.2024.06.033. Epub 2024 Jun 26. Mol Ther. 2024. PMID: 38937969 Free PMC article.
A Type II-B Cas9 nuclease with minimized off-targets and reduced chromosomal translocations in vivo.
Bestas B, Wimberger S, Degtev D, Madsen A, Rottner AK, Karlsson F, Naumenko S, Callahan M, Touza JL, Francescatto M, Möller CI, Badertscher L, Li S, Cerboni S, Selfjord N, Ericson E, Gordon E, Firth M, Chylinski K, Taheri-Ghahfarokhi A, Bohlooly-Y M, Snowden M, Pangalos M, Nuttall B, Akcakaya P, Sienski G, Maresca M. Bestas B, et al. Among authors: firth m. Nat Commun. 2023 Sep 6;14(1):5474. doi: 10.1038/s41467-023-41240-7. Nat Commun. 2023. PMID: 37673883 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. Among authors: firth m. 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.
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. Among authors: firth m. Nat Commun. 2023 Aug 14;14(1):4761. doi: 10.1038/s41467-023-40344-4. Nat Commun. 2023. PMID: 37580318 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.
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. Among authors: firth m. J Extracell Vesicles. 2022 May;11(5):e12225. doi: 10.1002/jev2.12225. J Extracell Vesicles. 2022. PMID: 35585651 Free PMC article.
129 results