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316 results

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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: maresca 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.
Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype.
Bjursell M, Porritt MJ, Ericson E, Taheri-Ghahfarokhi A, Clausen M, Magnusson L, Admyre T, Nitsch R, Mayr L, Aasehaug L, Seeliger F, Maresca M, Bohlooly-Y M, Wiseman J. Bjursell M, et al. Among authors: maresca m. EBioMedicine. 2018 Mar;29:104-111. doi: 10.1016/j.ebiom.2018.02.015. Epub 2018 Feb 19. EBioMedicine. 2018. PMID: 29500128 Free PMC article.
A CRISP(e)R view on kidney organoids allows generation of an induced pluripotent stem cell-derived kidney model for drug discovery.
Boreström C, Jonebring A, Guo J, Palmgren H, Cederblad L, Forslöw A, Svensson A, Söderberg M, Reznichenko A, Nyström J, Patrakka J, Hicks R, Maresca M, Valastro B, Collén A. Boreström C, et al. Among authors: maresca m. Kidney Int. 2018 Dec;94(6):1099-1110. doi: 10.1016/j.kint.2018.05.003. Epub 2018 Jul 31. Kidney Int. 2018. PMID: 30072040 Free article.
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: maresca m. 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.
In vivo genome and base editing of a human PCSK9 knock-in hypercholesterolemic mouse model.
Carreras A, Pane LS, Nitsch R, Madeyski-Bengtson K, Porritt M, Akcakaya P, Taheri-Ghahfarokhi A, Ericson E, Bjursell M, Perez-Alcazar M, Seeliger F, Althage M, Knöll R, Hicks R, Mayr LM, Perkins R, Lindén D, Borén J, Bohlooly-Y M, Maresca M. Carreras A, et al. Among authors: maresca m. BMC Biol. 2019 Jan 15;17(1):4. doi: 10.1186/s12915-018-0624-2. BMC Biol. 2019. PMID: 30646909 Free PMC article.
Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq.
Wienert B, Wyman SK, Richardson CD, Yeh CD, Akcakaya P, Porritt MJ, Morlock M, Vu JT, Kazane KR, Watry HL, Judge LM, Conklin BR, Maresca M, Corn JE. Wienert B, et al. Among authors: maresca m. Science. 2019 Apr 19;364(6437):286-289. doi: 10.1126/science.aav9023. Epub 2019 Apr 18. Science. 2019. PMID: 31000663 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: maresca m. Nat Commun. 2021 Jan 21;12(1):497. doi: 10.1038/s41467-020-20810-z. Nat Commun. 2021. PMID: 33479216 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: maresca 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.
316 results