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Page 1
Long-term lineage commitment in haematopoietic stem cell gene therapy.
Calabria A, Spinozzi G, Cesana D, Buscaroli E, Benedicenti F, Pais G, Gazzo F, Scala S, Lidonnici MR, Scaramuzza S, Albertini A, Esposito S, Tucci F, Canarutto D, Omrani M, De Mattia F, Dionisio F, Giannelli S, Marktel S, Fumagalli F, Calbi V, Cenciarelli S, Ferrua F, Gentner B, Caravagna G, Ciceri F, Naldini L, Ferrari G, Aiuti A, Montini E. Calabria A, et al. Among authors: canarutto d. Nature. 2024 Dec;636(8041):162-171. doi: 10.1038/s41586-024-08250-x. Epub 2024 Oct 23. Nature. 2024. PMID: 39442556 Free PMC article.
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, Gasperini S, Galimberti S, Silvani P, Filisetti C, Darin S, Forni G, Miglietta S, Santi L, Facchini M, Corti A, Fumagalli F, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Recupero S, Canarutto D, Doglio M, Tedesco L, Volpi N, Rovelli A, la Marca G, Valsecchi MG, Zancan S, Ciceri F, Naldini L, Baldoli C, Parini R, Gentner B, Aiuti A, Bernardo ME. Consiglieri G, et al. Among authors: canarutto d. Sci Transl Med. 2024 May;16(745):eadi8214. doi: 10.1126/scitranslmed.adi8214. Epub 2024 May 1. Sci Transl Med. 2024. PMID: 38691622 Clinical Trial.
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: canarutto d. 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.
Author Correction: Genotoxic effects of base and prime editing in human hematopoietic stem cells.
Fiumara M, Ferrari S, Omer-Javed A, Beretta S, Albano L, Canarutto D, Varesi A, Gaddoni C, Brombin C, Cugnata F, Zonari E, Naldini MM, Barcella M, Gentner B, Merelli I, Naldini L. Fiumara M, et al. Among authors: canarutto d. Nat Biotechnol. 2024 Jun;42(6):986. doi: 10.1038/s41587-024-02142-1. Nat Biotechnol. 2024. PMID: 38273067 Free PMC article. No abstract available.
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. 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.
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: canarutto d. 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.
Genotoxic effects of base and prime editing in human hematopoietic stem cells.
Fiumara M, Ferrari S, Omer-Javed A, Beretta S, Albano L, Canarutto D, Varesi A, Gaddoni C, Brombin C, Cugnata F, Zonari E, Naldini MM, Barcella M, Gentner B, Merelli I, Naldini L. Fiumara M, et al. Among authors: canarutto d. Nat Biotechnol. 2024 Jun;42(6):877-891. doi: 10.1038/s41587-023-01915-4. Epub 2023 Sep 7. Nat Biotechnol. 2024. PMID: 37679541 Free PMC article.
Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.
Canarutto D, Oltolini C, Barzaghi F, Calbi V, Migliavacca M, Tucci F, Gallo V, Consiglieri G, Ferrua F, Recupero S, Cervi MC, Al-Mousa H, Pituch-Noworolska A, Tassan Din C, Scarpellini P, Silvani P, Fossati C, Casiraghi M, Cirillo DM, Castagna A, Bernardo ME, Aiuti A, Cicalese MP. Canarutto D, et al. Biomedicines. 2023 Jun 24;11(7):1809. doi: 10.3390/biomedicines11071809. Biomedicines. 2023. PMID: 37509449 Free PMC article.
27 results