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7,064 results

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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: su l. 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: su l. 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.
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: su l. 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: su l. Sci Transl Med. 2017 Jan 11;9(372):eaah3480. doi: 10.1126/scitranslmed.aah3480. Sci Transl Med. 2017. PMID: 28077679
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency.
De Ravin SS, Liu S, Sweeney CL, Brault J, Whiting-Theobald N, Ma M, Liu T, Choi U, Lee J, O'Brien SA, Quackenbush P, Estwick T, Karra A, Docking E, Kwatemaa N, Guo S, Su L, Sun Z, Zhou S, Puck J, Cowan MJ, Notarangelo LD, Kang E, Malech HL, Wu X. De Ravin SS, et al. Among authors: su l. Nat Commun. 2022 Jun 28;13(1):3710. doi: 10.1038/s41467-022-31344-x. Nat Commun. 2022. PMID: 35764638 Free PMC article. Clinical Trial.
Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model.
Uchida N, Stasula U, Demirci S, Germino-Watnick P, Hinds M, Le A, Chu R, Berg A, Liu X, Su L, Wu X, Krouse AE, Linde NS, Bonifacino A, Hong SG, Dunbar CE, Lanieri L, Bhat A, Palchaudhuri R, Bennet B, Hoban M, Bertelsen K, Olson LM, Donahue RE, Tisdale JF. Uchida N, et al. Among authors: su l. Nat Commun. 2023 Oct 12;14(1):6291. doi: 10.1038/s41467-023-41153-5. Nat Commun. 2023. PMID: 37828021 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: su l. PLoS One. 2014 Jul 21;9(7):e101423. doi: 10.1371/journal.pone.0101423. eCollection 2014. PLoS One. 2014. PMID: 25047265 Free PMC article.
IKKα inactivation promotes Kras-initiated lung adenocarcinoma development through disrupting major redox regulatory pathways.
Song NY, Zhu F, Wang Z, Willette-Brown J, Xi S, Sun Z, Su L, Wu X, Ma B, Nussinov R, Xia X, Schrump DS, Johnson PF, Karin M, Hu Y. Song NY, et al. Among authors: su l. Proc Natl Acad Sci U S A. 2018 Jan 23;115(4):E812-E821. doi: 10.1073/pnas.1717520115. Epub 2018 Jan 8. Proc Natl Acad Sci U S A. 2018. PMID: 29311298 Free PMC article.
7,064 results