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Page 1
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo.
Cesana D, Ranzani M, Volpin M, Bartholomae C, Duros C, Artus A, Merella S, Benedicenti F, Sergi Sergi L, Sanvito F, Brombin C, Nonis A, Serio CD, Doglioni C, von Kalle C, Schmidt M, Cohen-Haguenauer O, Naldini L, Montini E. Cesana D, et al. Among authors: brombin c. Mol Ther. 2014 Apr;22(4):774-85. doi: 10.1038/mt.2014.3. Epub 2014 Jan 20. Mol Ther. 2014. PMID: 24441399 Free PMC article.
Niacin-mediated Tace activation ameliorates CMT neuropathies with focal hypermyelination.
Bolino A, Piguet F, Alberizzi V, Pellegatta M, Rivellini C, Guerrero-Valero M, Noseda R, Brombin C, Nonis A, D'Adamo P, Taveggia C, Previtali SC. Bolino A, et al. Among authors: brombin c. EMBO Mol Med. 2016 Dec 1;8(12):1438-1454. doi: 10.15252/emmm.201606349. Print 2016 Dec. EMBO Mol Med. 2016. PMID: 27799291 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: brombin c. 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: brombin c. 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.
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.
Milani M, Canepari C, Liu T, Biffi M, Russo F, Plati T, Curto R, Patarroyo-White S, Drager D, Visigalli I, Brombin C, Albertini P, Follenzi A, Ayuso E, Mueller C, Annoni A, Naldini L, Cantore A. Milani M, et al. Among authors: brombin c. Nat Commun. 2022 May 4;13(1):2454. doi: 10.1038/s41467-022-30102-3. Nat Commun. 2022. PMID: 35508619 Free PMC article.
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: brombin c. 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.
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: brombin c. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638 Free PMC article.
60 results