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High-dose enzyme replacement therapy in murine Hurler syndrome.
Ou L, Herzog T, Koniar BL, Gunther R, Whitley CB. Ou L, et al. Among authors: whitley cb. Mol Genet Metab. 2014 Feb;111(2):116-22. doi: 10.1016/j.ymgme.2013.09.008. Epub 2013 Sep 19. Mol Genet Metab. 2014. PMID: 24100243 Free PMC article.
Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.
Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C, Hayashi RJ, Shenoy S, Sillence D, Tiller GE, Dudek ME, van Royen-Kerkhof A, Wraith JE, Woodard P, Young GA, Wulffraat N, Whitley CB, Peters C. Grewal SS, et al. Among authors: whitley cb. Genet Med. 2005 Feb;7(2):143-6. doi: 10.1097/01.gim.0000154299.22120.6a. Genet Med. 2005. PMID: 15714083 Free article. Clinical Trial.
Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.
Aronovich EL, Bell JB, Belur LR, Gunther R, Koniar B, Erickson DC, Schachern PA, Matise I, McIvor RS, Whitley CB, Hackett PB. Aronovich EL, et al. Among authors: whitley cb. J Gene Med. 2007 May;9(5):403-15. doi: 10.1002/jgm.1028. J Gene Med. 2007. PMID: 17407189 Free PMC article.
192 results