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Page 1
Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report.
Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB. Engel BC, et al. Among authors: kohn db. Blood. 2007 Jan 15;109(2):503-6. doi: 10.1182/blood-2006-06-031476. Epub 2006 Sep 14. Blood. 2007. PMID: 16973956 Free PMC article.
Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion.
Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB. Carbonaro DA, et al. Among authors: kohn db. Blood. 2008 Jun 15;111(12):5745-54. doi: 10.1182/blood-2007-08-103663. Epub 2008 Mar 20. Blood. 2008. PMID: 18356486 Free PMC article.
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB. Candotti F, et al. Among authors: kohn db. Blood. 2012 Nov 1;120(18):3635-46. doi: 10.1182/blood-2012-02-400937. Epub 2012 Sep 11. Blood. 2012. PMID: 22968453 Free PMC article. Clinical Trial.
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: kohn db. 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.
Gene therapy outpaces haplo for SCID-X1.
Kohn DB. Kohn DB. Blood. 2015 Jun 4;125(23):3521-2. doi: 10.1182/blood-2015-04-641720. Blood. 2015. PMID: 26045591 Free 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: kohn db. 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.
360 results