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Page 1
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: jacob a. 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.
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: jacob a. Sci Transl Med. 2017 Oct 11;9(411):eaan0820. doi: 10.1126/scitranslmed.aan0820. Sci Transl Med. 2017. PMID: 29021165
Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking.
Ferrari S, Jacob A, Beretta S, Unali G, Albano L, Vavassori V, Cittaro D, Lazarevic D, Brombin C, Cugnata F, Kajaste-Rudnitski A, Merelli I, Genovese P, Naldini L. Ferrari S, et al. Among authors: jacob a. Nat Biotechnol. 2020 Nov;38(11):1298-1308. doi: 10.1038/s41587-020-0551-y. Epub 2020 Jun 29. Nat Biotechnol. 2020. PMID: 32601433 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. Among authors: jacob a. Nat Protoc. 2021 Jun;16(6):2991-3025. doi: 10.1038/s41596-021-00529-x. Epub 2021 May 24. Nat Protoc. 2021. PMID: 34031609
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. Among authors: jacob a. 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.
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo.
Asperti C, Canarutto D, Porcellini S, Sanvito F, Cecere F, Vavassori V, Ferrari S, Rovelli E, Albano L, Jacob A, Sergi Sergi L, Montaldo E, Ferrua F, González-Granado LI, Lougaris V, Badolato R, Finocchi A, Villa A, Radrizzani M, Naldini L. Asperti C, et al. Among authors: jacob a. Mol Ther Methods Clin Dev. 2023 Aug 23;30:546-557. doi: 10.1016/j.omtm.2023.08.020. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693944 Free PMC article.
Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1.
Canarutto D, Asperti C, Vavassori V, Porcellini S, Rovelli E, Paulis M, Ferrari S, Varesi A, Fiumara M, Jacob A, Sergi Sergi L, Visigalli I, Ferrua F, González-Granado LI, Lougaris V, Finocchi A, Villa A, Radrizzani M, Naldini L. Canarutto D, et al. Among authors: jacob a. EMBO J. 2023 Dec 1;42(23):e114188. doi: 10.15252/embj.2023114188. Epub 2023 Nov 2. EMBO J. 2023. PMID: 37916874 Free PMC article.
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Castiello MC, Brandas C, Ferrari S, Porcellini S, Sacchetti N, Canarutto D, Draghici E, Merelli I, Barcella M, Pelosi G, Vavassori V, Varesi A, Jacob A, Scala S, Basso Ricci L, Paulis M, Strina D, Di Verniere M, Sergi Sergi L, Serafini M, Holland SM, Bergerson JRE, De Ravin SS, Malech HL, Pala F, Bosticardo M, Brombin C, Cugnata F, Calzoni E, Crooks GM, Notarangelo LD, Genovese P, Naldini L, Villa A. Castiello MC, et al. Among authors: jacob a. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638
1,468 results