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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: wu x. 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.
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: wu x. 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.
ZNF367 inhibits cancer progression and is targeted by miR-195.
Jain M, Zhang L, Boufraqech M, Liu-Chittenden Y, Bussey K, Demeure MJ, Wu X, Su L, Pacak K, Stratakis CA, Kebebew E. Jain M, et al. Among authors: wu x. PLoS One. 2014 Jul 21;9(7):e101423. doi: 10.1371/journal.pone.0101423. eCollection 2014. PLoS One. 2014. PMID: 25047265 Free PMC article.
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.
De Ravin SS, Wu X, Moir S, Anaya-O'Brien S, Kwatemaa N, Littel P, Theobald N, Choi U, Su L, Marquesen M, Hilligoss D, Lee J, Buckner CM, Zarember KA, O'Connor G, McVicar D, Kuhns D, Throm RE, Zhou S, Notarangelo LD, Hanson IC, Cowan MJ, Kang E, Hadigan C, Meagher M, Gray JT, Sorrentino BP, Malech HL, Kardava L. De Ravin SS, et al. Among authors: wu x. Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856. Sci Transl Med. 2016. PMID: 27099176 Free PMC article. Clinical Trial.
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: wu x. Sci Transl Med. 2017 Jan 11;9(372):eaah3480. doi: 10.1126/scitranslmed.aah3480. Sci Transl Med. 2017. PMID: 28077679
Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease.
Merling RK, Kuhns DB, Sweeney CL, Wu X, Burkett S, Chu J, Lee J, Koontz S, Di Pasquale G, Afione SA, Chiorini JA, Kang EM, Choi U, De Ravin SS, Malech HL. Merling RK, et al. Among authors: wu x. Blood Adv. 2016 Dec 28;1(4):270-278. doi: 10.1182/bloodadvances.2016001214. eCollection 2017 Jan 10. Blood Adv. 2016. PMID: 29296942 Free PMC article.
Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque.
Espinoza DA, Fan X, Yang D, Cordes SF, Truitt LL, Calvo KR, Yabe IM, Demirci S, Hope KJ, Hong SG, Krouse A, Metzger M, Bonifacino A, Lu R, Uchida N, Tisdale JF, Wu X, DeRavin SS, Malech HL, Donahue RE, Wu C, Dunbar CE. Espinoza DA, et al. Among authors: wu x, wu c. Mol Ther. 2019 Jun 5;27(6):1074-1086. doi: 10.1016/j.ymthe.2019.04.003. Epub 2019 Apr 9. Mol Ther. 2019. PMID: 31023523 Free PMC article.
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