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Page 1
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.
Bzhilyanskaya V, Ma L, Liu S, Fox LR, Whittaker MN, Meis RJ, Choi U, Lawson A, Ma M, Theobald N, Burkett S, Sweeney CL, Lazzarotto CR, Tsai SQ, Lack JB, Wu X, Dahl GA, Malech HL, Kleinstiver BP, De Ravin SS. Bzhilyanskaya V, et al. Among authors: choi u. Sci Transl Med. 2024 Oct 16;16(769):eadj6779. doi: 10.1126/scitranslmed.adj6779. Epub 2024 Oct 16. Sci Transl Med. 2024. PMID: 39413163
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.
De Ravin SS, Reik A, Liu PQ, Li L, Wu X, Su L, Raley C, Theobald N, Choi U, Song AH, Chan A, Pearl JR, Paschon DE, Lee J, Newcombe H, Koontz S, Sweeney C, Shivak DA, Zarember KA, Peshwa MV, Gregory PD, Urnov FD, Malech HL. De Ravin SS, et al. Among authors: choi u. Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7. Nat Biotechnol. 2016. PMID: 26950749 Free PMC article.
CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.
De Ravin SS, Li L, Wu X, Choi U, Allen C, Koontz S, Lee J, Theobald-Whiting N, Chu J, Garofalo M, Sweeney C, Kardava L, Moir S, Viley A, Natarajan P, Su L, Kuhns D, Zarember KA, Peshwa MV, Malech HL. De Ravin SS, et al. Among authors: choi u. Sci Transl Med. 2017 Jan 11;9(372):eaah3480. doi: 10.1126/scitranslmed.aah3480. Sci Transl Med. 2017. PMID: 28077679
Gene Editing in Chronic Granulomatous Disease.
Sweeney CL, Merling RK, De Ravin SS, Choi U, Malech HL. Sweeney CL, et al. Among authors: choi u. Methods Mol Biol. 2019;1982:623-665. doi: 10.1007/978-1-4939-9424-3_36. Methods Mol Biol. 2019. PMID: 31172498
NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease.
De Ravin SS, Brault J, Meis RJ, Li L, Theobald N, Bonifacino AC, Lei H, Liu TQ, Koontz S, Corsino C, Zarakas MA, Desai JV, Clark AB, Choi U, Metzger ME, West K, Highfill SL, Kang E, Kuhns DB, Lionakis MS, Stroncek DF, Dunbar CE, Tisdale JF, Donahue RE, Dahl GA, Malech HL. De Ravin SS, et al. Among authors: choi u. Blood Adv. 2020 Dec 8;4(23):5976-5987. doi: 10.1182/bloodadvances.2020003224. Blood Adv. 2020. PMID: 33284949 Free PMC article.
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells.
De Ravin SS, Brault J, Meis RJ, Liu S, Li L, Pavel-Dinu M, Lazzarotto CR, Liu T, Koontz SM, Choi U, Sweeney CL, Theobald N, Lee G, Clark AB, Burkett SS, Kleinstiver BP, Porteus MH, Tsai S, Kuhns DB, Dahl GA, Headey S, Wu X, Malech HL. De Ravin SS, et al. Among authors: choi u. Blood. 2021 May 13;137(19):2598-2608. doi: 10.1182/blood.2020008503. Blood. 2021. PMID: 33623984 Free PMC article.
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.
Sweeney CL, Pavel-Dinu M, Choi U, Brault J, Liu T, Koontz S, Li L, Theobald N, Lee J, Bello EA, Wu X, Meis RJ, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Sweeney CL, et al. Among authors: choi u. Gene Ther. 2021 Jun;28(6):373-390. doi: 10.1038/s41434-021-00251-z. Epub 2021 Mar 12. Gene Ther. 2021. PMID: 33712802 Free PMC article.
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Brault J, Liu T, Bello E, Liu S, Sweeney CL, Meis RJ, Koontz S, Corsino C, Choi U, Vayssiere G, Bosticardo M, Dowdell K, Lazzarotto CR, Clark AB, Notarangelo LD, Ravell JC, Lenardo MJ, Kleinstiver BP, Tsai SQ, Wu X, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Among authors: choi u. Blood. 2021 Dec 30;138(26):2768-2780. doi: 10.1182/blood.2021011192. Blood. 2021. PMID: 34086870 Free PMC article.
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Brault J, et al. Among authors: choi u. Front Immunol. 2023 Jan 4;13:1067417. doi: 10.3389/fimmu.2022.1067417. eCollection 2022. Front Immunol. 2023. PMID: 36685559 Free PMC article.
363 results