Armbruster, N., Lattanzi, A., Jeavons, M., Van Wittenberghe, L., Gjata, B., Marais, T., Martin, S., Vignaud, A., Voit, T., Mavilio, F., Barkats, M., Buj-Bello, A. :
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
Mol Ther Methods Clin Dev, 2016 ; 3 (SP) : 16060
Publications (1181)
Seferian, A. M., Malfatti, E., Bosson, C., Pelletier, L., Taytard, J., Forin, V., Gidaro, T., Gargaun, E., Carlier, P., Faure, J., Romero, N. B., Rendu, J., Servais, L. :
Mild clinical presentation in KLHL40-related nemaline myopathy (NEM 8) Neuromuscul Disord, 2016 ; 26 (10) : 712-716
Mild clinical presentation in KLHL40-related nemaline myopathy (NEM 8) Neuromuscul Disord, 2016 ; 26 (10) : 712-716
Polesskaya, A., Pinna, G., Sassi, Y., Vandamme, M., Bigot, A., Mouly, V., Morozova, N., Harel-Bellan, A., Degerny, C. :
Post-transcriptional modulation of interleukin 8 by CNOT6L regulates skeletal muscle differentiation Biochim Biophys Acta, 2016 ; 1863 (2) : 263-70
Post-transcriptional modulation of interleukin 8 by CNOT6L regulates skeletal muscle differentiation Biochim Biophys Acta, 2016 ; 1863 (2) : 263-70
Dragin, N., Bismuth, J., Cizeron-Clairac, G., Biferi, M. G., Berthault, C., Serraf, A., Nottin, R., Klatzmann, D., Cumano, A., Barkats, M., Le Panse, R., Berrih-Aknin, S. :
Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases J Clin Invest, 2016 ; 126 (4) : 1525-37
Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases J Clin Invest, 2016 ; 126 (4) : 1525-37
Le Moing, A. G., Seferian, A. M., Moraux, A., Annoussamy, M., Dorveaux, E., Gasnier, E., Hogrel, J. Y., Voit, T., Vissiere, D., Servais, L. :
A Movement Monitor Based on Magneto-Inertial Sensors for Non-Ambulant Patients with Duchenne Muscular Dystrophy: A Pilot Study in Controlled Environment PLoS ONE, 2016 ; 11 (6) : e0156696
A Movement Monitor Based on Magneto-Inertial Sensors for Non-Ambulant Patients with Duchenne Muscular Dystrophy: A Pilot Study in Controlled Environment PLoS ONE, 2016 ; 11 (6) : e0156696
Carlier, P. G., Marty, B., Scheidegger, O., Loureiro de Sousa, P., Baudin, P. Y., Snezhko, E., Vlodavets, D. :
Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials J Neuromuscul Dis, 2016 ; 3 (1) : 1-28
Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials J Neuromuscul Dis, 2016 ; 3 (1) : 1-28
Allenbach, Y., Leroux, G., Suarez-Calvet, X., Preusse, C., Gallardo, E., Hervier, B., Rigolet, A., Hie, M., Pehl, D., Limal, N., Hufnagl, P., Zerbe, N., Meyer, A., Aouizerate, J., Uzunhan, Y., Maisonobe, T., Goebel, H. H., Benveniste, O., Stenzel, W., French Myositis, Network :
Dermatomyositis With or Without Anti-Melanoma Differentiation-Associated Gene 5 Antibodies: Common Interferon Signature but Distinct NOS2 Expression Am J Pathol, 2016 ; 186 (3) : 691-700
Dermatomyositis With or Without Anti-Melanoma Differentiation-Associated Gene 5 Antibodies: Common Interferon Signature but Distinct NOS2 Expression Am J Pathol, 2016 ; 186 (3) : 691-700
Alves, S., Marais, T., Biferi, M. G., Furling, D., Marinello, M., El Hachimi, K., Cartier, N., Ruberg, M., Stevanin, G., Brice, A., Barkats, M., Sittler, A. :
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins Mol Neurodegener, 2016 ; 11 (1) : 1-20
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins Mol Neurodegener, 2016 ; 11 (1) : 1-20
Vilmont, V., Cadot, B., Ouanounou, G., Gomes, E. R. :
A system for studying mechanisms of neuromuscular junction development and maintenance Development, 2016 ; 143 (13) : 2464-77
A system for studying mechanisms of neuromuscular junction development and maintenance Development, 2016 ; 143 (13) : 2464-77
Gargaun, E., Seferian, A. M., Cardas, R., Le Moing, A. G., Delanoe, C., Nectoux, J., Nelson, I., Bonne, G., Bihoreau, M. T., Deleuze, J. F., Boland, A., Masson, C., Servais, L., Gidaro, T. :
EGR2 mutation enhances phenotype spectrum of Dejerine-Sottas syndrome J Neurol, 2016 ; 263 (7) : 1456-8
EGR2 mutation enhances phenotype spectrum of Dejerine-Sottas syndrome J Neurol, 2016 ; 263 (7) : 1456-8
