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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Weber L, Frati G, Felix T, Hardouin G, Casini A, Wollenschlaeger C, Meneghini V, Masson C, De Cian A, Chalumeau A, Mavilio F, Amendola M, Andre-Schmutz I, Cereseto A, El Nemer W, Concordet JP, Giovannangeli C, Cavazzana M, Miccio A. Weber L, et al. Among authors: casini a. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
A highly specific SpCas9 variant is identified by in vivo screening in yeast.
Casini A, Olivieri M, Petris G, Montagna C, Reginato G, Maule G, Lorenzin F, Prandi D, Romanel A, Demichelis F, Inga A, Cereseto A. Casini A, et al. Nat Biotechnol. 2018 Mar;36(3):265-271. doi: 10.1038/nbt.4066. Epub 2018 Jan 29. Nat Biotechnol. 2018. PMID: 29431739 Free PMC article.
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
Ramadier S, Chalumeau A, Felix T, Othman N, Aknoun S, Casini A, Maule G, Masson C, De Cian A, Frati G, Brusson M, Concordet JP, Cavazzana M, Cereseto A, El Nemer W, Amendola M, Wattellier B, Meneghini V, Miccio A. Ramadier S, et al. Among authors: casini a. Mol Ther. 2022 Jan 5;30(1):145-163. doi: 10.1016/j.ymthe.2021.08.019. Epub 2021 Aug 19. Mol Ther. 2022. PMID: 34418541 Free PMC article.
An optimized SpCas9 high-fidelity variant for direct protein delivery.
Pedrazzoli E, Bianchi A, Umbach A, Amistadi S, Brusson M, Frati G, Ciciani M, Badowska KA, Arosio D, Miccio A, Cereseto A, Casini A. Pedrazzoli E, et al. Among authors: casini a. Mol Ther. 2023 Jul 5;31(7):2257-2265. doi: 10.1016/j.ymthe.2023.03.007. Epub 2023 Mar 10. Mol Ther. 2023. PMID: 36905119 Free article.
Eukaryotic-driven directed evolution of Cas9 nucleases.
Ruta GV, Ciciani M, Kheir E, Gentile MD, Amistadi S, Casini A, Cereseto A. Ruta GV, et al. Among authors: casini a. Genome Biol. 2024 Mar 25;25(1):79. doi: 10.1186/s13059-024-03215-9. Genome Biol. 2024. PMID: 38528620 Free PMC article.
741 results