Result Number | Material Type | Add to My Shelf Action | Record Details and Options |
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1 |
Material Type: Artigo
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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional OutcomesMendell, Jerry R. ; Sahenk, Zarife ; Al-Zaidy, Samiah ; Rodino-Klapac, Louise R. ; Lowes, Linda P. ; Alfano, Lindsay N. ; Berry, Katherine ; Miller, Natalie ; Yalvac, Mehmet ; Dvorchik, Igor ; Moore-Clingenpeel, Melissa ; Flanigan, Kevin M. ; Church, Kathleen ; Shontz, Kim ; Curry, Choumpree ; Lewis, Sarah ; McColly, Markus ; Hogan, Mark J. ; Kaspar, Brian K.Molecular therapy, 2017-04, Vol.25 (4), p.870-879 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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2 |
Material Type: Artigo
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Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon SkippingVulin, Adeline ; Barthélémy, Inès ; Goyenvalle, Aurélie ; Thibaud, Jean-Laurent ; Beley, Cyriaque ; Griffith, Graziella ; Benchaouir, Rachid ; le Hir, Maëva ; Unterfinger, Yves ; Lorain, Stéphanie ; Dreyfus, Patrick ; Voit, Thomas ; Carlier, Pierre ; Blot, Stéphane ; Garcia, LuisMolecular therapy, 2012-11, Vol.20 (11), p.2120-2133 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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3 |
Material Type: Artigo
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Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne PatientsLe Guiner, Caroline ; Montus, Marie ; Servais, Laurent ; Cherel, Yan ; Francois, Virginie ; Thibaud, Jean-Laurent ; Wary, Claire ; Matot, Béatrice ; Larcher, Thibaut ; Guigand, Lydie ; Dutilleul, Maeva ; Domenger, Claire ; Allais, Marine ; Beuvin, Maud ; Moraux, Amélie ; Le Duff, Johanne ; Devaux, Marie ; Jaulin, Nicolas ; Guilbaud, Mickaël ; Latournerie, Virginie ; Veron, Philippe ; Boutin, Sylvie ; Leborgne, Christian ; Desgue, Diana ; Deschamps, Jack-Yves ; Moullec, Sophie ; Fromes, Yves ; Vulin, Adeline ; Smith, Richard H ; Laroudie, Nicolas ; Barnay-Toutain, Frédéric ; Rivière, Christel ; Bucher, Stéphanie ; Le, Thanh-Hoa ; Delaunay, Nicolas ; Gasmi, Mehdi ; Kotin, Robert M ; Bonne, Gisèle ; Adjali, Oumeya ; Masurier, Carole ; Hogrel, Jean-Yves ; Carlier, Pierre ; Moullier, Philippe ; Voit, ThomasMolecular therapy, 2014-11, Vol.22 (11), p.1923-1935 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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4 |
Material Type: Artigo
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Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal MuscleToromanoff, Alice ; Adjali, Oumeya ; Larcher, Thibaut ; Hill, Marcelo ; Guigand, Lydie ; Chenuaud, Pierre ; Deschamps, Jack-Yves ; Gauthier, Olivier ; Blancho, Gilles ; Vanhove, Bernard ; Rolling, Fabienne ; Chérel, Yan ; Moullier, Philippe ; Anegon, Ignacio ; Le Guiner, CarolineMolecular therapy, 2010-01, Vol.18 (1), p.151-160 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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5 |
Material Type: Artigo
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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE ExpressionVidal, Patrice ; Pagliarani, Serena ; Colella, Pasqualina ; Costa Verdera, Helena ; Jauze, Louisa ; Gjorgjieva, Monika ; Puzzo, Francesco ; Marmier, Solenne ; Collaud, Fanny ; Simon Sola, Marcelo ; Charles, Severine ; Lucchiari, Sabrina ; van Wittenberghe, Laetitia ; Vignaud, Alban ; Gjata, Bernard ; Richard, Isabelle ; Laforet, Pascal ; Malfatti, Edoardo ; Mithieux, Gilles ; Rajas, Fabienne ; Comi, Giacomo Pietro ; Ronzitti, Giuseppe ; Mingozzi, FedericoMolecular therapy, 2018-03, Vol.26 (3), p.890-901 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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6 |
Material Type: Artigo
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Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular DystrophyGoyenvalle, Aurélie ; Wright, Jordan ; Babbs, Arran ; Wilkins, Vivienne ; Garcia, Luis ; Davies, Kay EMolecular therapy, 2012-06, Vol.20 (6), p.1212-1221 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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7 |
Material Type: Artigo
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Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy PatientsOusterout, David G ; Perez-Pinera, Pablo ; Thakore, Pratiksha I ; Kabadi, Ami M ; Brown, Matthew T ; Qin, Xiaoxia ; Fedrigo, Olivier ; Mouly, Vincent ; Tremblay, Jacques P ; Gersbach, Charles AMolecular therapy, 2013-09, Vol.21 (9), p.1718-1726 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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8 |
Material Type: Artigo
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The Rag2–Il2rb–Dmd– Mouse: a Novel Dystrophic and Immunodeficient Model to Assess Innovating Therapeutic Strategies for Muscular DystrophiesVallese, Denis ; Negroni, Elisa ; Duguez, Stéphanie ; Ferry, Arnaud ; Trollet, Capucine ; Aamiri, Ahmed ; Vosshenrich, Christian AJ ; Füchtbauer, Ernst-Martin ; Di Santo, James P ; Vitiello, Libero ; Butler-Browne, Gillian ; Mouly, VincentMolecular therapy, 2013-10, Vol.21 (10), p.1950-1957 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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9 |
Material Type: Artigo
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Cis-splicing and Translation of the Pre-Trans-splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans-splicingMonjaret, François ; Bourg, Nathalie ; Suel, Laurence ; Roudaut, Carinne ; Le Roy, Florence ; Richard, Isabelle ; Charton, KarineMolecular therapy, 2014-06, Vol.22 (6), p.1176-1187 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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10 |
Material Type: Artigo
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ASGCT 19th Annual Meeting: Author IndexMolecular therapy, 2016-04, Vol.24 Suppl 1 (Supplement 1), p.S305-S319 [Periódico revisado por pares]United States: Elsevier LimitedTexto completo disponível |