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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: genovese p. 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.
Targeted genome editing in human repopulating haematopoietic stem cells.
Genovese P, Schiroli G, Escobar G, Tomaso TD, Firrito C, Calabria A, Moi D, Mazzieri R, Bonini C, Holmes MC, Gregory PD, van der Burg M, Gentner B, Montini E, Lombardo A, Naldini L. Genovese P, et al. Nature. 2014 Jun 12;510(7504):235-240. doi: 10.1038/nature13420. Epub 2014 May 28. Nature. 2014. PMID: 24870228 Free PMC article.
NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease.
Mastaglio S, Genovese P, Magnani Z, Ruggiero E, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Rocchi M, Oliveira G, Escobar G, Casucci M, Gentner B, Spinelli A, Mondino A, Bondanza A, Vago L, Ponzoni M, Ciceri F, Holmes MC, Naldini L, Bonini C. Mastaglio S, et al. Among authors: genovese p. Blood. 2017 Aug 3;130(5):606-618. doi: 10.1182/blood-2016-08-732636. Epub 2017 Jun 21. Blood. 2017. PMID: 28637663 Free article.
Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients.
Diez B, Genovese P, Roman-Rodriguez FJ, Alvarez L, Schiroli G, Ugalde L, Rodriguez-Perales S, Sevilla J, Diaz de Heredia C, Holmes MC, Lombardo A, Naldini L, Bueren JA, Rio P. Diez B, et al. Among authors: genovese p. EMBO Mol Med. 2017 Nov;9(11):1574-1588. doi: 10.15252/emmm.201707540. EMBO Mol Med. 2017. PMID: 28899930 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: genovese p. Sci Transl Med. 2017 Oct 11;9(411):eaan0820. doi: 10.1126/scitranslmed.aan0820. Sci Transl Med. 2017. PMID: 29021165
Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells.
Petrillo C, Thorne LG, Unali G, Schiroli G, Giordano AMS, Piras F, Cuccovillo I, Petit SJ, Ahsan F, Noursadeghi M, Clare S, Genovese P, Gentner B, Naldini L, Towers GJ, Kajaste-Rudnitski A. Petrillo C, et al. Among authors: genovese p. Cell Stem Cell. 2018 Dec 6;23(6):820-832.e9. doi: 10.1016/j.stem.2018.10.008. Epub 2018 Nov 8. Cell Stem Cell. 2018. PMID: 30416070 Free PMC article.
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: genovese p. 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.
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: genovese p. 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.
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome.
Vavassori V, Mercuri E, Marcovecchio GE, Castiello MC, Schiroli G, Albano L, Margulies C, Buquicchio F, Fontana E, Beretta S, Merelli I, Cappelleri A, Rancoita PM, Lougaris V, Plebani A, Kanariou M, Lankester A, Ferrua F, Scanziani E, Cotta-Ramusino C, Villa A, Naldini L, Genovese P. Vavassori V, et al. Among authors: genovese p. EMBO Mol Med. 2021 Mar 5;13(3):e13545. doi: 10.15252/emmm.202013545. Epub 2021 Jan 21. EMBO Mol Med. 2021. PMID: 33475257 Free PMC article.
85 results