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In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy.
Choudhury SR, Fitzpatrick Z, Harris AF, Maitland SA, Ferreira JS, Zhang Y, Ma S, Sharma RB, Gray-Edwards HL, Johnson JA, Johnson AK, Alonso LC, Punzo C, Wagner KR, Maguire CA, Kotin RM, Martin DR, Sena-Esteves M. Choudhury SR, et al. Among authors: kotin rm. Mol Ther. 2016 Aug;24(7):1247-57. doi: 10.1038/mt.2016.84. Epub 2016 Apr 27. Mol Ther. 2016. PMID: 27117222 Free PMC article.
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: kotin rm. 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.
Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping.
Bish LT, Sleeper MM, Forbes SC, Wang B, Reynolds C, Singletary GE, Trafny D, Morine KJ, Sanmiguel J, Cecchini S, Virag T, Vulin A, Beley C, Bogan J, Wilson JM, Vandenborne K, Kornegay JN, Walter GA, Kotin RM, Garcia L, Sweeney HL. Bish LT, et al. Among authors: kotin rm. Mol Ther. 2012 Mar;20(3):580-9. doi: 10.1038/mt.2011.264. Epub 2011 Dec 6. Mol Ther. 2012. PMID: 22146342 Free PMC article.
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