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Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy.
Hurlbut GD, Ziegler RJ, Nietupski JB, Foley JW, Woodworth LA, Meyers E, Bercury SD, Pande NN, Souza DW, Bree MP, Lukason MJ, Marshall J, Cheng SH, Scheule RK. Hurlbut GD, et al. Among authors: souza dw. Mol Ther. 2010 Nov;18(11):1983-94. doi: 10.1038/mt.2010.175. Epub 2010 Aug 24. Mol Ther. 2010. PMID: 20736932 Free PMC article.
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.
Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao G, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC, Cheng SH. Ziegler RJ, et al. Among authors: souza dw. Mol Ther. 2004 Feb;9(2):231-40. doi: 10.1016/j.ymthe.2003.11.015. Mol Ther. 2004. PMID: 14759807 Free article.
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