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Base-editing mutagenesis maps alleles to tune human T cell functions.
Schmidt R, Ward CC, Dajani R, Armour-Garb Z, Ota M, Allain V, Hernandez R, Layeghi M, Xing G, Goudy L, Dorovskyi D, Wang C, Chen YY, Ye CJ, Shy BR, Gilbert LA, Eyquem J, Pritchard JK, Dodgson SE, Marson A. Schmidt R, et al. Nature. 2024 Jan;625(7996):805-812. doi: 10.1038/s41586-023-06835-6. Epub 2023 Dec 13. Nature. 2024. PMID: 38093011 Free PMC article.
High-yield genome engineering in primary cells using a hybrid ssDNA repair template and small-molecule cocktails.
Shy BR, Vykunta VS, Ha A, Talbot A, Roth TL, Nguyen DN, Pfeifer WG, Chen YY, Blaeschke F, Shifrut E, Vedova S, Mamedov MR, Chung JJ, Li H, Yu R, Wu D, Wolf J, Martin TG, Castro CE, Ye L, Esensten JH, Eyquem J, Marson A. Shy BR, et al. Nat Biotechnol. 2023 Apr;41(4):521-531. doi: 10.1038/s41587-022-01418-8. Epub 2022 Aug 25. Nat Biotechnol. 2023. PMID: 36008610 Free PMC article.
Lentiviral Gene Therapy for Artemis-Deficient SCID.
Cowan MJ, Yu J, Facchino J, Fraser-Browne C, Sanford U, Kawahara M, Dara J, Long-Boyle J, Oh J, Chan W, Chag S, Broderick L, Chellapandian D, Decaluwe H, Golski C, Hu D, Kuo CY, Miller HK, Petrovic A, Currier R, Hilton JF, Punwani D, Dvorak CC, Malech HL, McIvor RS, Puck JM. Cowan MJ, et al. N Engl J Med. 2022 Dec 22;387(25):2344-2355. doi: 10.1056/NEJMoa2206575. N Engl J Med. 2022. PMID: 36546626 Free PMC article. Clinical Trial.
Systematic decoding of cis gene regulation defines context-dependent control of the multi-gene costimulatory receptor locus in human T cells.
Mowery CT, Freimer JW, Chen Z, Casaní-Galdón S, Umhoefer JM, Arce MM, Gjoni K, Daniel B, Sandor K, Gowen BG, Nguyen V, Simeonov DR, Garrido CM, Curie GL, Schmidt R, Steinhart Z, Satpathy AT, Pollard KS, Corn JE, Bernstein BE, Ye CJ, Marson A. Mowery CT, et al. Nat Genet. 2024 Jun;56(6):1156-1167. doi: 10.1038/s41588-024-01743-5. Epub 2024 May 29. Nat Genet. 2024. PMID: 38811842 Free PMC article.
Poor T-cell receptor β repertoire diversity early posttransplant for severe combined immunodeficiency predicts failure of immune reconstitution.
Delmonte OM, Castagnoli R, Yu J, Dvorak CC, Cowan MJ, Dávila Saldaña BJ, De Ravin SS, Mamcarz E, Chang CK, Daley SR, Griffith LM, Notarangelo LD, Puck JM. Delmonte OM, et al. J Allergy Clin Immunol. 2022 Mar;149(3):1113-1119. doi: 10.1016/j.jaci.2021.07.029. Epub 2021 Aug 9. J Allergy Clin Immunol. 2022. PMID: 34384841 Free PMC article.
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