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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: cugnata f. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638
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: cugnata f. Nat Biotechnol. 2024 Jan 25. doi: 10.1038/s41587-024-02142-1. Online ahead of print. Nat Biotechnol. 2024. PMID: 38273067 No abstract available.
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: cugnata f. Nat Biotechnol. 2023 Sep 7. doi: 10.1038/s41587-023-01915-4. Online ahead of print. Nat Biotechnol. 2023. PMID: 37679541
34 results