Search Results - "Montus, Marie"

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors by Boutin, Sylvie, Monteilhet, Virginie, Veron, Philippe, Leborgne, Christian, Benveniste, Olivier, Montus, Marie Françoise, Masurier, Carole

“…Adeno-associated viruses (AAVs) are small, nonenveloped single-stranded DNA viruses that require helper viruses to facilitate efficient replication. Despite…”
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A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8 by Monteilhet, Virginie, Saheb, Samir, Boutin, Sylvie, Leborgne, Christian, Veron, Philippe, Montus, Marie-Françoise, Moullier, Philippe, Benveniste, Olivier, Masurier, Carole

“…Adeno-associated viruses (AAV) are small, nonenveloped single-stranded DNA viruses which require helper viruses to facilitate efficient replication. These…”
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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy by Le Guiner, Caroline, Servais, Laurent, Montus, Marie, Larcher, Thibaut, Fraysse, Bodvaël, Moullec, Sophie, Allais, Marine, François, Virginie, Dutilleul, Maeva, Malerba, Alberto, Koo, Taeyoung, Thibaut, Jean-Laurent, Matot, Béatrice, Devaux, Marie, Le Duff, Johanne, Deschamps, Jack-Yves, Barthelemy, Inès, Blot, Stéphane, Testault, Isabelle, Wahbi, Karim, Ederhy, Stéphane, Martin, Samia, Veron, Philippe, Georger, Christophe, Athanasopoulos, Takis, Masurier, Carole, Mingozzi, Federico, Carlier, Pierre, Gjata, Bernard, Hogrel, Jean-Yves, Adjali, Oumeya, Mavilio, Fulvio, Voit, Thomas, Moullier, Philippe, Dickson, George

“…Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly…”
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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 by 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

“…Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available. Our…”
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Distinctive serum miRNA profile in mouse models of striated muscular pathologies by Vignier, Nicolas, Amor, Fatima, Fogel, Paul, Duvallet, Angélique, Poupiot, Jérôme, Charrier, Sabine, Arock, Michel, Montus, Marie, Nelson, Isabelle, Richard, Isabelle, Carrier, Lucie, Servais, Laurent, Voit, Thomas, Bonne, Gisèle, Israeli, David

“…Biomarkers are critically important for disease diagnosis and monitoring. In particular, close monitoring of disease evolution is eminently required for the…”
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Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy by Gentil, Christel, Le Guiner, Caroline, Falcone, Sestina, Hogrel, Jean-Yves, Peccate, Cécile, Lorain, Stéphanie, Benkhelifa-Ziyyat, Sofia, Guigand, Lydie, Montus, Marie, Servais, Laurent, Voit, Thomas, Piétri-Rouxel, France

“…At present, the clinically most advanced strategy to treat Duchenne muscular dystrophy (DMD) is the exon-skipping strategy. Whereas antisense…”
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An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress by Israeli, David, Cosette, Jérémie, Corre, Guillaume, Amor, Fatima, Poupiot, Jérôme, Stockholm, Daniel, Montus, Marie, Gjata, Bernard, Richard, Isabelle

“…Sarcoglycanopathies are rare autosomic limb girdle muscular dystrophies caused by mutations in one of the genes coding for sarcoglycans. Sarcoglycans form a…”
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Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models by Marangoni, Francesco, Bosticardo, Marita, Charrier, Sabine, Draghici, Elena, Locci, Michela, Scaramuzza, Samantha, Panaroni, Cristina, Ponzoni, Maurilio, Sanvito, Francesca, Doglioni, Claudio, Liabeuf, Marie, Gjata, Bernard, Montus, Marie, Siminovitch, Katherine, Aiuti, Alessandro, Naldini, Luigi, Dupré, Loïc, Roncarolo, Maria Grazia, Galy, Anne, Villa, Anna

“…Wiskott–Aldrich Syndrome (WAS) is a life-threatening X-linked disease characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancies. Gene…”
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RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect by Domenger, Claire, Allais, Marine, François, Virginie, Léger, Adrien, Lecomte, Emilie, Montus, Marie, Servais, Laurent, Voit, Thomas, Moullier, Philippe, Audic, Yann, Le Guiner, Caroline

“…Non-coding uridine-rich small nuclear RNAs (UsnRNAs) have emerged in recent years as effective tools for exon skipping for the treatment of Duchenne muscular…”
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Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy by Mariot, Virginie, Le Guiner, Caroline, Barthélémy, Inès, Montus, Marie, Blot, Stéphane, Torelli, Silvia, Morgan, Jennifer, Muntoni, Francesco, Voit, Thomas, Dumonceaux, Julie

“…Recently, several promising treatments have emerged for neuromuscular disorders, highlighting the need for robust biomarkers for monitoring therapeutic…”
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Quantitative NMRI and NMRS identify augmented disease progression after loss of ambulation in forearms of boys with Duchenne muscular dystrophy by Wary, Claire, Azzabou, Noura, Giraudeau, Céline, Le Louër, Julien, Montus, Marie, Voit, Thomas, Servais, Laurent, Carlier, Pierre

“…Quantitative NMRI and 31P NMRS indices are reported in the forearms of 24 patients with Duchenne muscular dystrophy (DMD) (6–18 years, 14 non‐ambulant)…”
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A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C by Herson, Serge, Hentati, Faycal, Rigolet, Aude, Behin, Anthony, Romero, Norma B., Leturcq, France, Laforêt, Pascal, Maisonobe, Thierry, Amouri, Rim, Haddad, Hafedh, Audit, Muriel, Montus, Marie, Masurier, Carole, Gjata, Bernard, Georger, Christophe, Cheraï, Mustapha, Carlier, Pierre, Hogrel, Jean-Yves, Herson, Ariane, Allenbach, Yves, Lemoine, François M., Klatzmann, David, Sweeney, H. Lee, Mulligan, Richard C., Eymard, Bruno, Caizergues, Didier, Voït, Thomas, Benveniste, Olivier

“…γ-Sarcoglycanopathy or limb girdle muscular dystrophy type 2C is an untreatable disease caused by autosomal recessively inherited mutations of the…”
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AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis by Buj-Bello, Anna, Fougerousse, Françoise, Schwab, Yannick, Messaddeq, Nadia, Spehner, Danièle, Pierson, Christopher R., Durand, Muriel, Kretz, Christine, Danos, Olivier, Douar, Anne-Marie, Beggs, Alan H., Schultz, Patrick, Montus, Marie, Denèfle, Patrice, Mandel, Jean-Louis

“…Myotubular myopathy (XLMTM, OMIM 310400) is a severe congenital muscular disease due to mutations in the myotubularin gene (MTM1) and characterized by the…”
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143. Off-Target Analysis of a rAAV-U7snRNA Vector Used for the Treatment of Duchenne Patients By Exon Skipping by Domenger, Claire, Lardenois, Aurélie, Allais, Marine, François, Virginie, Montus, Marie, Servais, Laurent, Voit, Thomas, Moullier, Philippe, Le Guiner, Caroline

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503. Adeno-Associated Virus Vector (AAV) Microdystrophin Gene Therapy Prolongs Survival and Restores Muscle Function in the Canine Model of Duchenne Muscular Dystrophy (DMD) by Guiner, Caroline Le, Servais, Laurent, Montus, Marie, Larcher, Thibaut, Fraysse, Bodvael, Moullec, Sophie, Koo, Taeyoung, Malerba, Alberto, Bec, Christine Le, Hebben, Matthias, Masurier, Carole, Mingozzi, Federico, Adjali, Oumeya, Carlier, Pierre, Hogrel, Jean-Yves, Gjata, Bernard, Cherel, Yan, Athanasopoulos, Takis, Mavilio, Fulvio, Voit, Thomas, Moullier, Philippe, Dickson, George

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Corrigendum to “Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models” by Marangoni, Francesco, Bosticardo, Marita, Charrier, Sabine, Draghici, Elena, Locci, Michela, Scaramuzza, Samantha, Panaroni, Cristina, Ponzoni, Maurilio, Sanvito, Francesca, Doglioni, Claudio, Liabeuf, Marie, Gjata, Bernard, Montus, Marie, Siminovitch, Katherine, Aiuti, Alessandro, Naldini, Luigi, Dupré, Loïc, Roncarolo, Maria Grazia, Galy, Anne, Villa, Anna

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AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis by Buj-Bello, Anna, Fougerousse, Françoise, Schwab, Yannick, Messaddeq, Nadia, Spehner, Danièle, Pierson, Christopher R., Durand, Muriel, Kretz, Christine, Danos, Olivier, Douar, Anne-Marie, Beggs, Alan H., Schultz, Patrick, Montus, Marie, Denèfle, Patrice, Mandel, Jean-Louis

“…Myotubular myopathy (XLMTM, OMIM 310400) is a severe congenital muscular disease due to mutations in the myotubularin gene (MTM1) and characterized by the…”
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