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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. Among authors: campo fernandez b. 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.
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. 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.
IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System.
Puig-Saus C, Parisi G, Garcia-Diaz A, Krystofinski PE, Sandoval S, Zhang R, Champhekar AS, McCabe J, Cheung-Lau GC, Truong NA, Vega-Crespo A, Komenan MDS, Pang J, Macabali MH, Saco JD, Goodwin JL, Bolon B, Seet CS, Montel-Hagen A, Crooks GM, Hollis RP, Campo-Fernandez B, Bischof D, Cornetta K, Gschweng EH, Adelson C, Nguyen A, Yang L, Witte ON, Baltimore D, Comin-Anduix B, Kohn DB, Wang X, Cabrera P, Kaplan-Lefko PJ, Berent-Maoz B, Ribas A. Puig-Saus C, et al. Clin Cancer Res. 2019 Feb 1;25(3):1000-1011. doi: 10.1158/1078-0432.CCR-18-0963. Epub 2018 Nov 8. Clin Cancer Res. 2019. PMID: 30409823 Free PMC article.
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: campo fernandez b. 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.
24 results