Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy von Bourg, Nathalie, Vu Hong, Ai, Lostal, William, Jaber, Abbass, Guerchet, Nicolas, Tanniou, Guillaume, Bordier, Fanny, Bertil-Froidevaux, Emilie, Georger, Christophe, Daniele, Nathalie, Richard, Isabelle, Israeli, David

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Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back von Buscara, Laurine, Gross, David-Alexandre, Daniele, Nathalie

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Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver von Jauze, Louisa, Vie, Mallaury, Miagoux, Quentin, Rossiaud, Lucille, Vidal, Patrice, Montalvo-Romeral, Valle, Saliba, Hanadi, Jarrige, Margot, Polveche, Helene, Nozi, Justine, Le Brun, Pierre-Romain, Bocchialini, Luca, Francois, Amandine, Cosette, Jérémie, Rouillon, Jérémy, Collaud, Fanny, Bordier, Fanny, Bertil-Froidevaux, Emilie, Georger, Christophe, van Wittenberghe, Laetitia, Miranda, Adeline, Daniele, Nathalie F, Gross, David-Alexandre, Hoch, Lucile, Nissan, Xavier, Ronzitti, Giuseppe

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AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice von Colella, Pasqualina, Sellier, Pauline, Costa Verdera, Helena, Puzzo, Francesco, van Wittenberghe, Laetitia, Guerchet, Nicolas, Daniele, Nathalie, Gjata, Bernard, Marmier, Solenne, Charles, Severine, Simon Sola, Marcelo, Ragone, Isabella, Leborgne, Christian, Collaud, Fanny, Mingozzi, Federico

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Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice von Danièle, Nathalie, Moal, Christelle, Julien, Laura, Marinello, Martina, Jamet, Thibaud, Martin, Samia, Vignaud, Alban, Lawlor, Michael W, Buj-Bello, Ana

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Cardiac ankyrin repeat protein is a marker of skeletal muscle pathological remodelling von Laure, Lydie, Suel, Laurence, Roudaut, Carinne, Bourg, Nathalie, Ouali, Ahmed, Bartoli, Marc, Richard, Isabelle, Danièle, Nathalie

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A new pathway encompassing calpain 3 and its newly identified substrate cardiac ankyrin repeat protein is involved in the regulation of the nuclear factor-[kappa]B pathway in skele... von Laure, Lydie, Daniele, Nathalie, Suel, Laurence, Marchand, Sylvie, Aubert, Sophie, Bourg, Nathalie, Roudaut, Carinne, Duguez, Stephanie, Bartoli, Marc, Richard, Isabelle

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new pathway encompassing calpain 3 and its newly identified substrate cardiac ankyrin repeat protein is involved in the regulation of the nuclear factor-κB pathway in skeletal musc... von Laure, Lydie, Danièle, Nathalie, Suel, Laurence, Marchand, Sylvie, Aubert, Sophie, Bourg, Nathalie, Roudaut, Carinne, Duguez, Stéphanie, Bartoli, Marc, Richard, Isabelle

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Ins and outs of therapy in limb girdle muscular dystrophies von Danièle, Nathalie, Richard, Isabelle, Bartoli, Marc

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Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies von Charton, Karine, Danièle, Nathalie, Vihola, Anna, Roudaut, Carinne, Gicquel, Evelyne, Monjaret, François, Tarrade, Anne, Sarparanta, Jaakko, Udd, Bjarne, Richard, Isabelle

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A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III von Gardin, Antoine, Rouillon, Jérémy, Montaivo-Romera, Valle, Rossiaud, Lucille, Vidal, Patrice, Launay, Romain, Vie, Mallaury, Benchekroun, Youssef Krimi, Cosette, Jérémie, Bertin, Bérangére, La Bella, Tiziana, Dubreuil, Guillaume, Nozi, Justine, Jauze, Louisa, Fragnoud, Romain, Daniele, Nathalie, Van Wittenberghe, Laetitia, Esque, Jérémy, André, Isabelle, Nissan, Xavier, Hoch, Lucile, Ronzitti, Giuseppe

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A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III von Gardin, Antoine, Rouillon, Jérémy, Montalvo-Romeral, Valle, Rossiaud, Lucille, Vidal, Patrice, Launay, Romain, Vie, Mallaury, Krimi Benchekroun, Youssef, Cosette, Jérémie, Bertin, Bérangère, La Bella, Tiziana, Dubreuil, Guillaume, Nozi, Justine, Jauze, Louisa, Fragnoud, Romain, Daniele, Nathalie, Van Wittenberghe, Laetitia, Esque, Jérémy, André, Isabelle, Nissan, Xavier, Hoch, Lucile, Ronzitti, Giuseppe

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Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase von Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, Van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Collaud, Fanny, Charles, Severine, Sola, Marcelo Simon, Boyer, Olivier, Krijnse-Locker, Jacomina, Ronzitti, Giuseppe, Colella, Pasqualina, Mingozzi, Federico

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NF-κB-dependent expression of the antiapoptotic factor c-FLIP is regulated by calpain 3, the protein involved in limb-girdle muscular dystrophy type 2A von Benayoun, Béatrice, Baghdiguian, Stephen, Lajmanovich, Alicia, Bartoli, Marc, Daniele, Nathalie, Gicquel, Evelyne, Bourg, Nathalie, Raynaud, Fabrice, Pasquier, Marie-Anne, Suel, Laurence, Lochmuller, Hanns, Lefranc, Gérard, Richard, Isabelle

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Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase von Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Collaud, Fanny, Charles, Severine, Sola, Marcelo Simon, Boyer, Olivier, Krijnse-Locker, Jacomina, Ronzitti, Giuseppe, Colella, Pasqualina, Mingozzi, Federico

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Phosphatidylinositol 3-Kinase Translocates onto Liver Endoplasmic Reticulum and May Account for the Inhibition of Glucose-6-phosphatase during Refeeding von Daniele, Nathalie, Rajas, Fabienne, Payrastre, Bernard, Mauco, Gérard, Zitoun, Carine, Mithieux, Gilles

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PI 3-kinase p110β: a new target for antithrombotic therapy von Jackson, Shaun P, Schoenwaelder, Simone M, Goncalves, Isaac, Nesbitt, Warwick S, Yap, Cindy L, Wright, Christine E, Kenche, Vijaya, Anderson, Karen E, Dopheide, Sacha M, Yuan, Yuping, Sturgeon, Sharelle A, Prabaharan, Hishani, Thompson, Philip E, Smith, Gregg D, Shepherd, Peter R, Daniele, Nathalie, Kulkarni, Suhasini, Abbott, Belinda, Saylik, Dilek, Jones, Catherine, Lu, Lucy, Giuliano, Simon, Hughan, Sascha C, Angus, James A, Robertson, Alan D, Salem, Hatem H

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Liver Microsomal Glucose-6-phosphatase Is Competitively Inhibited by the Lipid Products of Phosphatidylinositol 3-Kinase von Mithieux, Gilles, Daniele, Nathalie, Payrastre, Bernard, Zitoun, Carine

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Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa−/−mice von Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, Van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Krijnse-Locker, Jacomine, Ronzitti, Giuseppe, Colella, Pasqualina, Mingozzi, Federico

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Evidence for functional redundancy of class IA PI3K isoforms in insulin signalling von Chaussade, Claire, Rewcastle, Gordon W, Kendall, Jackie D, Denny, William A, Cho, Kitty, Grønning, Line M, Chong, Mei Ling, Anagnostou, Sasha H, Jackson, Shaun P, Daniele, Nathalie, Shepherd, Peter R

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