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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
In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene.
Larochelle A, Choi U, Shou Y, Naumann N, Loktionova NA, Clevenger JR, Krouse A, Metzger M, Donahue RE, Kang E, Stewart C, Persons D, Malech HL, Dunbar CE, Sorrentino BP. Larochelle A, et al. Among authors: choi u. J Clin Invest. 2009 Jul;119(7):1952-63. doi: 10.1172/JCI37506. Epub 2009 Jun 8. J Clin Invest. 2009. PMID: 19509470 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.
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
Merling RK, Sweeney CL, Chu J, Bodansky A, Choi U, Priel DL, Kuhns DB, Wang H, Vasilevsky S, De Ravin SS, Winkler T, Dunbar CE, Zou J, Zarember KA, Gallin JI, Holland SM, Malech HL. Merling RK, et al. Among authors: choi u. Mol Ther. 2015 Jan;23(1):147-57. doi: 10.1038/mt.2014.195. Epub 2014 Oct 7. Mol Ther. 2015. PMID: 25288370 Free PMC article.
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.
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.
Enhancers are major targets for murine leukemia virus vector integration.
De Ravin SS, Su L, Theobald N, Choi U, Macpherson JL, Poidinger M, Symonds G, Pond SM, Ferris AL, Hughes SH, Malech HL, Wu X. De Ravin SS, et al. Among authors: choi u. J Virol. 2014 Apr;88(8):4504-13. doi: 10.1128/JVI.00011-14. Epub 2014 Feb 5. J Virol. 2014. PMID: 24501411 Free PMC article.
343 results