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Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/beta -microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells.
Brenner S, Whiting-Theobald NL, Linton GF, Holmes KL, Anderson-Cohen M, Kelly PF, Vanin EF, Pilon AM, Bodine DM, Horwitz ME, Malech HL. Brenner S, et al. Among authors: vanin ef. Blood. 2003 Oct 15;102(8):2789-97. doi: 10.1182/blood-2002-05-1482. Epub 2003 Jun 26. Blood. 2003. PMID: 12829597 Free article.
Gene transfer into hematopoietic cells.
Nienhuis AW, Bertran J, Hargrove P, Vanin E, Yang Y. Nienhuis AW, et al. Stem Cells. 1997;15 Suppl 1:123-34. doi: 10.1002/stem.5530150816. Stem Cells. 1997. PMID: 9368332 Review.
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