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Items: 10


Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome.

Kuo CY, Long JD, Campo-Fernandez B, de Oliveira S, Cooper AR, Romero Z, Hoban MD, Joglekar AV, Lill GR, Kaufman ML, Fitz-Gibbon S, Wang X, Hollis RP, Kohn DB.

Cell Rep. 2018 May 29;23(9):2606-2616. doi: 10.1016/j.celrep.2018.04.103.


Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells.

Long J, Hoban MD, Cooper AR, Kaufman ML, Kuo CY, Campo-Fernandez B, Lumaquin D, Hollis RP, Wang X, Kohn DB, Romero Z.

Mol Ther. 2018 Feb 7;26(2):468-479. doi: 10.1016/j.ymthe.2017.11.001. Epub 2017 Nov 9.


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.

Mol Ther. 2016 Sep;24(9):1561-9. doi: 10.1038/mt.2016.148. Epub 2016 Jul 29.


Analyzing CRISPR genome-editing experiments with CRISPResso.

Pinello L, Canver MC, Hoban MD, Orkin SH, Kohn DB, Bauer DE, Yuan GC.

Nat Biotechnol. 2016 Jul 12;34(7):695-7. doi: 10.1038/nbt.3583. No abstract available.


A genome editing primer for the hematologist.

Hoban MD, Bauer DE.

Blood. 2016 May 26;127(21):2525-35. doi: 10.1182/blood-2016-01-678151. Epub 2016 Apr 6. Review.


Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells.

Hoban MD, Romero Z, Cost GJ, Mendel M, Holmes M, Kohn DB.

Curr Protoc Stem Cell Biol. 2016 Feb 3;36:5B.4.1-10. doi: 10.1002/9780470151808.sc05b04s36. Review.


Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.

Hoban MD, Orkin SH, Bauer DE.

Blood. 2016 Feb 18;127(7):839-48. doi: 10.1182/blood-2015-09-618587. Epub 2016 Jan 12. Review.


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.

Mol Ther Methods Clin Dev. 2015 Apr 22;2:15012. doi: 10.1038/mtm.2015.12. eCollection 2015.


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.

Blood. 2015 Apr 23;125(17):2597-604. doi: 10.1182/blood-2014-12-615948. Epub 2015 Mar 2.


Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors.

Joglekar AV, Stein L, Ho M, Hoban MD, Hollis RP, Kohn DB.

Hum Gene Ther. 2014 Jul;25(7):599-608. doi: 10.1089/hum.2013.211. Epub 2014 Apr 2.

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