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1
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes

Mendell, 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 Inc

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2
Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping
Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping
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Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

Vulin, 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, Luis

Molecular therapy, 2012-11, Vol.20 (11), p.2120-2133 [Periódico revisado por pares]

United States: Elsevier Inc

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3
Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients
Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients
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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, 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, Thomas

Molecular therapy, 2014-11, Vol.22 (11), p.1923-1935 [Periódico revisado por pares]

United States: Elsevier Inc

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4
Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle
Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle
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Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle

Toromanoff, 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, Caroline

Molecular therapy, 2010-01, Vol.18 (1), p.151-160 [Periódico revisado por pares]

United States: Elsevier Inc

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5
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression

Vidal, 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, Federico

Molecular therapy, 2018-03, Vol.26 (3), p.890-901 [Periódico revisado por pares]

United States: Elsevier Inc

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6
Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy
Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy
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Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy

Goyenvalle, Aurélie ; Wright, Jordan ; Babbs, Arran ; Wilkins, Vivienne ; Garcia, Luis ; Davies, Kay E

Molecular therapy, 2012-06, Vol.20 (6), p.1212-1221 [Periódico revisado por pares]

United States: Elsevier Inc

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7
Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
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Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

Ousterout, 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 A

Molecular therapy, 2013-09, Vol.21 (9), p.1718-1726 [Periódico revisado por pares]

United States: Elsevier Inc

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8
Cis-splicing and Translation of the Pre-Trans-splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans-splicing
Cis-splicing and Translation of the Pre-Trans-splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans-splicing
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Cis-splicing and Translation of the Pre-Trans-splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans-splicing

Monjaret, François ; Bourg, Nathalie ; Suel, Laurence ; Roudaut, Carinne ; Le Roy, Florence ; Richard, Isabelle ; Charton, Karine

Molecular therapy, 2014-06, Vol.22 (6), p.1176-1187 [Periódico revisado por pares]

United States: Elsevier Inc

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9
The Rag2–Il2rb–Dmd– Mouse: a Novel Dystrophic and Immunodeficient Model to Assess Innovating Therapeutic Strategies for Muscular Dystrophies
The Rag2–Il2rb–Dmd– Mouse: a Novel Dystrophic and Immunodeficient Model to Assess Innovating Therapeutic Strategies for Muscular Dystrophies
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The Rag2–Il2rb–Dmd– Mouse: a Novel Dystrophic and Immunodeficient Model to Assess Innovating Therapeutic Strategies for Muscular Dystrophies

Vallese, 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, Vincent

Molecular therapy, 2013-10, Vol.21 (10), p.1950-1957 [Periódico revisado por pares]

United States: Elsevier Inc

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10
A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model
A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model
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A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model

Biferi, Maria Grazia ; Cohen-Tannoudji, Mathilde ; Cappelletto, Ambra ; Giroux, Benoit ; Roda, Marianne ; Astord, Stéphanie ; Marais, Thibaut ; Bos, Corinne ; Voit, Thomas ; Ferry, Arnaud ; Barkats, Martine

Molecular therapy, 2017-09, Vol.25 (9), p.2038-2052 [Periódico revisado por pares]

United States: Elsevier Inc

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