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Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing.
McAuley GE, Yiu G, Chang PC, Newby GA, Campo-Fernandez B, Fitz-Gibbon ST, Wu X, Kang SL, Garibay A, Butler J, Christian V, Wong RL, Everette KA, Azzun A, Gelfer H, Seet CS, Narendran A, Murguia-Favela L, Romero Z, Wright N, Liu DR, Crooks GM, Kohn DB. McAuley GE, et al. Among authors: romero z. Cell. 2023 Mar 30;186(7):1398-1416.e23. doi: 10.1016/j.cell.2023.02.027. Epub 2023 Mar 20. Cell. 2023. PMID: 36944331 Free PMC article.
CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.
Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz-Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, Hollis RP, Kohn DB. Hoban MD, et al. Among authors: romero z. Mol Ther. 2016 Sep;24(9):1561-9. doi: 10.1038/mt.2016.148. Epub 2016 Jul 29. Mol Ther. 2016. PMID: 27406980 Free PMC article.
Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.
Romero Z, Lomova A, Said S, Miggelbrink A, Kuo CY, Campo-Fernandez B, Hoban MD, Masiuk KE, Clark DN, Long J, Sanchez JM, Velez M, Miyahira E, Zhang R, Brown D, Wang X, Kurmangaliyev YZ, Hollis RP, Kohn DB. Romero Z, et al. Mol Ther. 2019 Aug 7;27(8):1389-1406. doi: 10.1016/j.ymthe.2019.05.014. Epub 2019 May 24. Mol Ther. 2019. PMID: 31178391 Free PMC article.
High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.
Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. Magis W, et al. Among authors: romero zg. iScience. 2022 May 10;25(6):104374. doi: 10.1016/j.isci.2022.104374. eCollection 2022 Jun 17. iScience. 2022. PMID: 35633935 Free PMC article.
36 results