Glycine receptor subunit-β-deficiency in a mouse model of spasticity results in attenuated physical performance, growth, and muscle strength

por Rivares, Cintia, Vignaud, Alban, Noort, Wendy, Koopmans, Bastijn, Loos, Maarten, Kalinichev, Mikhail, Jaspers, Richard T.
Publicado 2022

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

por Poletti, Valentina, Charrier, Sabine, Corre, Guillaume, Gjata, Bernard, Vignaud, Alban, Zhang, Fang, Rothe, Michael, Schambach, Axel, Gaspar, H. Bobby, Thrasher, Adrian J., Mavilio, Fulvio
Publicado 2018

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

por Armbruster, Nicole, Lattanzi, Annalisa, Jeavons, Matthieu, Van Wittenberghe, Laetitia, Gjata, Bernard, Marais, Thibaut, Martin, Samia, Vignaud, Alban, Voit, Thomas, Mavilio, Fulvio, Barkats, Martine, Buj-Bello, Ana
Publicado 2016

Increased Muscle Stress-Sensitivity Induced by Selenoprotein N Inactivation in Mouse: A Mammalian Model for SEPN1-Related Myopathy

por Rederstorff, Mathieu, Castets, Perrine, Arbogast, Sandrine, Lainé, Jeanne, Vassilopoulos, Stéphane, Beuvin, Maud, Dubourg, Odile, Vignaud, Alban, Ferry, Arnaud, Krol, Alain, Allamand, Valérie, Guicheney, Pascale, Ferreiro, Ana, Lescure, Alain
Publicado 2011

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

por Fitzpatrick, Zachary, Leborgne, Christian, Barbon, Elena, Masat, Elisa, Ronzitti, Giuseppe, van Wittenberghe, Laetitia, Vignaud, Alban, Collaud, Fanny, Charles, Séverine, Simon Sola, Marcelo, Jouen, Fabienne, Boyer, Olivier, Mingozzi, Federico
Publicado 2018

Recombinant botulinum neurotoxin serotype A1 in vivo characterization

por Périer, Cindy, Martin, Vincent, Cornet, Sylvie, Favre‐Guilmard, Christine, Rocher, Marie‐Noelle, Bindler, Julien, Wagner, Stéphanie, Andriambeloson, Emile, Rudkin, Brian, Marty, Rudy, Vignaud, Alban, Beard, Matthew, Lezmi, Stephane, Kalinichev, Mikhail
Publicado 2021

Impaired Adaptive Response to Mechanical Overloading in Dystrophic Skeletal Muscle

por Joanne, Pierre, Hourdé, Christophe, Ochala, Julien, Caudéran, Yvain, Medja, Fadia, Vignaud, Alban, Mouisel, Etienne, Hadj-Said, Wahiba, Arandel, Ludovic, Garcia, Luis, Goyenvalle, Aurélie, Mounier, Rémi, Zibroba, Daria, Sakamato, Kei, Butler-Browne, Gillian, Agbulut, Onnik, Ferry, Arnaud
Publicado 2012

Correction: Impaired Adaptive Response to Mechanical Overloading in Dystrophic Skeletal Muscle

por Joanne, Pierre, Hourdé, Christophe, Ochala, Julien, Caudéran, Yvain, Medja, Fadia, Vignaud, Alban, Mouisel, Etienne, Hadj-Said, Wahiba, Arandel, Ludovic, Garcia, Luis, Goyenvalle, Aurélie, Mounier, Rémi, Zibroba, Daria, Sakamato, Kei, Butler-Browne, Gillian, Agbulut, Onnik, Ferry, Arnaud
Publicado 2013

Molecular, Physiological, and Motor Performance Defects in DMSXL Mice Carrying >1,000 CTG Repeats from the Human DM1 Locus

por Huguet, Aline, Medja, Fadia, Nicole, Annie, Vignaud, Alban, Guiraud-Dogan, Céline, Ferry, Arnaud, Decostre, Valérie, Hogrel, Jean-Yves, Metzger, Friedrich, Hoeflich, Andreas, Baraibar, Martin, Gomes-Pereira, Mário, Puymirat, Jack, Bassez, Guillaume, Furling, Denis, Munnich, Arnold, Gourdon, Geneviève
Publicado 2012

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

por Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, Laetitia, Vignaud, Alban, Veron, Philippe, Bosma, Piter J, Muro, Andres F, Mingozzi, Federico
Publicado 2016

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

por Meliani, Amine, Boisgerault, Florence, Hardet, Romain, Marmier, Solenne, Collaud, Fanny, Ronzitti, Giuseppe, Leborgne, Christian, Costa Verdera, Helena, Simon Sola, Marcelo, Charles, Severine, Vignaud, Alban, van Wittenberghe, Laetitia, Manni, Giorgia, Christophe, Olivier, Fallarino, Francesca, Roy, Christopher, Michaud, Alicia, Ilyinskii, Petr, Kishimoto, Takashi Kei, Mingozzi, Federico
Publicado 2018

Muscle inactivation of mTOR causes metabolic and dystrophin defects leading to severe myopathy

por Risson, Valérie, Mazelin, Laetitia, Roceri, Mila, Sanchez, Hervé, Moncollin, Vincent, Corneloup, Claudine, Richard-Bulteau, Hélène, Vignaud, Alban, Baas, Dominique, Defour, Aurélia, Freyssenet, Damien, Tanti, Jean-François, Le-Marchand-Brustel, Yannick, Ferrier, Bernard, Conjard-Duplany, Agnès, Romanino, Klaas, Bauché, Stéphanie, Hantaï, Daniel, Mueller, Matthias, Kozma, Sara C., Thomas, George, Rüegg, Markus A., Ferry, Arnaud, Pende, Mario, Bigard, Xavier, Koulmann, Nathalie, Schaeffer, Laurent, Gangloff, Yann-Gaël
Publicado 2009