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Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells.
Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, Calabria A, Vavassori V, Cipriani C, Castiello MC, Esposito S, Brombin C, Cugnata F, Adjali O, Ayuso E, Merelli I, Villa A, Di Micco R, Kajaste-Rudnitski A, Montini E, Penaud-Budloo M, Naldini L. Ferrari S, et al. Cell Stem Cell. 2022 Oct 6;29(10):1428-1444.e9. doi: 10.1016/j.stem.2022.09.001. Cell Stem Cell. 2022. PMID: 36206730 Free PMC article.
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.
Schiroli G, Ferrari S, Conway A, Jacob A, Capo V, Albano L, Plati T, Castiello MC, Sanvito F, Gennery AR, Bovolenta C, Palchaudhuri R, Scadden DT, Holmes MC, Villa A, Sitia G, Lombardo A, Genovese P, Naldini L. Schiroli G, et al. Among authors: ferrari s. Sci Transl Med. 2017 Oct 11;9(411):eaan0820. doi: 10.1126/scitranslmed.aan0820. Sci Transl Med. 2017. PMID: 29021165
Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response.
Schiroli G, Conti A, Ferrari S, Della Volpe L, Jacob A, Albano L, Beretta S, Calabria A, Vavassori V, Gasparini P, Salataj E, Ndiaye-Lobry D, Brombin C, Chaumeil J, Montini E, Merelli I, Genovese P, Naldini L, Di Micco R. Schiroli G, et al. Among authors: ferrari s. Cell Stem Cell. 2019 Apr 4;24(4):551-565.e8. doi: 10.1016/j.stem.2019.02.019. Epub 2019 Mar 21. Cell Stem Cell. 2019. PMID: 30905619 Free PMC article.
BAR-Seq clonal tracking of gene-edited cells.
Ferrari S, Beretta S, Jacob A, Cittaro D, Albano L, Merelli I, Naldini L, Genovese P. Ferrari S, et al. Nat Protoc. 2021 Jun;16(6):2991-3025. doi: 10.1038/s41596-021-00529-x. Epub 2021 May 24. Nat Protoc. 2021. PMID: 34031609
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs.
Crippa S, Conti A, Vavassori V, Ferrari S, Beretta S, Rivis S, Bosotti R, Scala S, Pirroni S, Jofra-Hernandez R, Santi L, Basso-Ricci L, Merelli I, Genovese P, Aiuti A, Naldini L, Di Micco R, Bernardo ME. Crippa S, et al. Among authors: ferrari s. Mol Ther. 2023 Jan 4;31(1):230-248. doi: 10.1016/j.ymthe.2022.08.011. Epub 2022 Aug 17. Mol Ther. 2023. PMID: 35982622 Free PMC article.
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs.
Crippa S, Conti A, Vavassori V, Ferrari S, Beretta S, Rivis S, Bosotti R, Scala S, Pirroni S, Jofra-Hernandez R, Santi L, Basso-Ricci L, Merelli I, Genovese P, Aiuti A, Naldini L, Di Micco R, Bernardo ME. Crippa S, et al. Among authors: ferrari s. Mol Ther. 2022 Oct 5;30(10):3333. doi: 10.1016/j.ymthe.2022.09.005. Epub 2022 Sep 11. Mol Ther. 2022. PMID: 36096132 Free PMC article. No abstract available.
2,621 results