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β-globin gene transfer to human bone marrow for sickle cell disease.
Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, Kempert P, Shupien S, Abdel-Azim H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB. Romero Z, et al. J Clin Invest. 2013 Jul 1;123(8):3317-30. doi: 10.1172/JCI67930. Online ahead of print. J Clin Invest. 2013. PMID: 23863630 Free PMC article.
Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells.
Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A. Urbinati F, et al. Among authors: romero z. Exp Hematol. 2015 May;43(5):346-351. doi: 10.1016/j.exphem.2015.01.009. Epub 2015 Feb 12. Exp Hematol. 2015. PMID: 25681747 Free PMC article.
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, Paschon DE, Zhang L, Rebar EJ, Wilber A, Wang X, Gregory PD, Holmes MC, Reik A, Hollis RP, Kohn DB. Hoban MD, et al. Among authors: romero z. Blood. 2015 Apr 23;125(17):2597-604. doi: 10.1182/blood-2014-12-615948. Epub 2015 Mar 2. Blood. 2015. PMID: 25733580 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.
Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.
Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, Kohn DB. Urbinati F, et al. Among authors: romero z. Cytotherapy. 2017 Sep;19(9):1096-1112. doi: 10.1016/j.jcyt.2017.06.002. Epub 2017 Jul 18. Cytotherapy. 2017. PMID: 28733131
37 results