Selected publications
- Brusson M, Chalumeau A, Martinucci P, et al. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies. Mol Ther Nucleic Acids. 2023;32:229-246. doi:10.1016/j.omtn.2023.03.012
- Pedrazzoli E, Bianchi A, Umbach A, Amistadi S, Brusson M, Frati G, Ciciani M, Badowska KA, Arosio D, Miccio A, Cereseto A, Casini A. An optimized SpCas9 high-fidelity variant for direct protein delivery. Mol Ther. 2023 Jul 5;31(7):2257-2265. doi: 10.1016/j.ymthe.2023.03.007.
- 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. Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression. Nat Commun. 2022 Nov 4;13(1):6618. doi: 10.1038/s41467-022-34493-1.
- Antoniou P, Miccio A, Brusson M. Base and Prime Editing Technologies for Blood Disorders. Front Genome Ed. 2021 Jan 28;3:618406. doi: 10.3389/fgeed.2021.618406.
- Amendola M, Brusson M, Miccio A. CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy. Stem Cells Transl Med. 2022 Oct 21;11(10):1003-1009. doi: 10.1093/stcltm/szac064.