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Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Brusson M, Chalumeau A, Martinucci P, Romano O, Felix T, Poletti V, Scaramuzza S, Ramadier S, Masson C, Ferrari G, Mavilio F, Cavazzana M, Amendola M, Miccio A. Brusson M, et al. Mol Ther Nucleic Acids. 2023 Mar 22;32:229-246. doi: 10.1016/j.omtn.2023.03.012. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37090420 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: brusson m. 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.
Genome editing approaches to β-hemoglobinopathies.
Brusson M, Miccio A. Brusson M, et al. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1. Prog Mol Biol Transl Sci. 2021. PMID: 34175041
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.
Antoniou P, Hardouin G, Martinucci P, Frati G, Felix T, Chalumeau A, Fontana L, Martin J, Masson C, Brusson M, Maule G, Rosello M, Giovannangeli C, Abramowski V, de Villartay JP, Concordet JP, Del Bene F, El Nemer W, Amendola M, Cavazzana M, Cereseto A, Romano O, Miccio A. Antoniou P, et al. Among authors: brusson m. Nat Commun. 2022 Nov 4;13(1):6618. doi: 10.1038/s41467-022-34493-1. Nat Commun. 2022. PMID: 36333351 Free PMC article.
Base and Prime Editing Technologies for Blood Disorders.
Antoniou P, Miccio A, Brusson M. Antoniou P, et al. Among authors: brusson m. Front Genome Ed. 2021 Jan 28;3:618406. doi: 10.3389/fgeed.2021.618406. eCollection 2021. Front Genome Ed. 2021. PMID: 34713251 Free PMC article. Review.
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: brusson m. 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.
Endothelial Cells Harbouring the JAK2V617F Mutation Display Pro-Adherent and Pro-Thrombotic Features.
Guadall A, Lesteven E, Letort G, Awan Toor S, Delord M, Pognant D, Brusson M, Verger E, Maslah N, Giraudier S, Larghero J, Vanneaux V, Chomienne C, El Nemer W, Cassinat B, Kiladjian JJ. Guadall A, et al. Among authors: brusson m. Thromb Haemost. 2018 Sep;118(9):1586-1599. doi: 10.1055/s-0038-1667015. Epub 2018 Aug 13. Thromb Haemost. 2018. PMID: 30103245
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