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Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.
Le Guiner C, Montus M, Servais L, Cherel Y, Francois V, Thibaud JL, Wary C, Matot B, Larcher T, Guigand L, Dutilleul M, Domenger C, Allais M, Beuvin M, Moraux A, Le Duff J, Devaux M, Jaulin N, Guilbaud M, Latournerie V, Veron P, Boutin S, Leborgne C, Desgue D, Deschamps JY, Moullec S, Fromes Y, Vulin A, Smith RH, Laroudie N, Barnay-Toutain F, Rivière C, Bucher S, Le TH, Delaunay N, Gasmi M, Kotin RM, Bonne G, Adjali O, Masurier C, Hogrel JY, Carlier P, Moullier P, Voit T. Le Guiner C, et al. Among authors: smith rh. Mol Ther. 2014 Nov;22(11):1923-35. doi: 10.1038/mt.2014.151. Epub 2014 Aug 4. Mol Ther. 2014. PMID: 25200009 Free PMC article.
Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency.
Nasimuzzaman M, Lynn D, Ernst R, Beuerlein M, Smith RH, Shrestha A, Cross S, Link K, Lutzko C, Nordling D, Russell DW, Larochelle A, Malik P, Van der Loo JC. Nasimuzzaman M, et al. Among authors: smith rh. Mol Ther Methods Clin Dev. 2016 Mar 16;3:16004. doi: 10.1038/mtm.2016.4. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27722179 Free PMC article.
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