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Page 1
PABPN1 gene therapy for oculopharyngeal muscular dystrophy.
Malerba A, Klein P, Bachtarzi H, Jarmin SA, Cordova G, Ferry A, Strings V, Espinoza MP, Mamchaoui K, Blumen SC, St Guily JL, Mouly V, Graham M, Butler-Browne G, Suhy DA, Trollet C, Dickson G. Malerba A, et al. Among authors: suhy da. Nat Commun. 2017 Mar 31;8:14848. doi: 10.1038/ncomms14848. Nat Commun. 2017. PMID: 28361972 Free PMC article.
BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy.
Strings-Ufombah V, Malerba A, Kao SC, Harbaran S, Roth F, Cappellari O, Lu-Nguyen N, Takahashi K, Mukadam S, Kilfoil G, Kloth C, Roelvink P, Dickson G, Trollet C, Suhy D. Strings-Ufombah V, et al. Mol Ther Nucleic Acids. 2021 Feb 18;24:67-78. doi: 10.1016/j.omtn.2021.02.017. eCollection 2021 Jun 4. Mol Ther Nucleic Acids. 2021. PMID: 33738139 Free PMC article.
Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model.
Suhy DA, Kao SC, Mao T, Whiteley L, Denise H, Souberbielle B, Burdick AD, Hayes K, Wright JF, Lavender H, Roelvink P, Kolykhalov A, Brady K, Moschos SA, Hauck B, Zelenaia O, Zhou S, Scribner C, High KA, Renison SH, Corbau R. Suhy DA, et al. Mol Ther. 2012 Sep;20(9):1737-49. doi: 10.1038/mt.2012.119. Epub 2012 Jun 26. Mol Ther. 2012. PMID: 22735378 Free PMC article.