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Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.
Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. Poletti V, et al. Among authors: urbinati f. Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014. eCollection 2018 Dec 14. Mol Ther Methods Clin Dev. 2018. PMID: 30533448 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: urbinati f. 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.
The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells.
Romero Z, Campo-Fernandez B, Wherley J, Kaufman ML, Urbinati F, Cooper AR, Hoban MD, Baldwin KM, Lumaquin D, Wang X, Senadheera S, Hollis RP, Kohn DB. Romero Z, et al. Among authors: urbinati f. Mol Ther Methods Clin Dev. 2015 Apr 22;2:15012. doi: 10.1038/mtm.2015.12. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26029723 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: urbinati f. 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.
40 results