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Base Editing of Human Hematopoietic Stem Cells.
Zeng J, Casirati G, Nguyen MA, Genovese P, Bauer DE. Zeng J, et al. Methods Mol Biol. 2023;2606:43-62. doi: 10.1007/978-1-0716-2879-9_5. Methods Mol Biol. 2023. PMID: 36592307 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. 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.
Epitope editing enables targeted immunotherapy of acute myeloid leukaemia.
Casirati G, Cosentino A, Mucci A, Salah Mahmoud M, Ugarte Zabala I, Zeng J, Ficarro SB, Klatt D, Brendel C, Rambaldi A, Ritz J, Marto JA, Pellin D, Bauer DE, Armstrong SA, Genovese P. Casirati G, et al. Nature. 2023 Sep;621(7978):404-414. doi: 10.1038/s41586-023-06496-5. Epub 2023 Aug 30. Nature. 2023. PMID: 37648862 Free PMC article.