Mearini, G., Stimpel, D., Kramer, E., Geertz, B., Braren, I., Gedicke-Hornung, C., Precigout, G., Muller, O. J., Katus, H. A., Eschenhagen, T., Voit, T., Garcia, L., Lorain, S., Carrier, L. :
Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy
Mol Ther Nucleic Acids, 2013 ; 2 : e102
Publications (1181)
Relaix, F., Demignon, J., Laclef, C., Pujol, J., Santolini, M., Niro, C., Lagha, M., Rocancourt, D., Buckingham, M., Maire, P. :
Six homeoproteins directly activate myod expression in the gene regulatory networks that control early myogenesis PLoS Genet, 2013 ; 9 (4) : e1003425
Six homeoproteins directly activate myod expression in the gene regulatory networks that control early myogenesis PLoS Genet, 2013 ; 9 (4) : e1003425
Wahbi, K., Behin, A., Becane, H. M., Leturcq, F., Cossee, M., Laforet, P., Stojkovic, T., Carlier, P., Toussaint, M., Gaxotte, V., Cluzel, P., Eymard, B., Duboc, D. :
Dilated cardiomyopathy in patients with mutations in anoctamin 5 Int J Cardiol, 2013 ; 168 (1) : 76-9
Dilated cardiomyopathy in patients with mutations in anoctamin 5 Int J Cardiol, 2013 ; 168 (1) : 76-9
Mercier, S., Toutain, A., Toussaint, A., Raynaud, M., de Barace, C., Marcorelles, P., Pasquier, L., Blayau, M., Espil, C., Parent, P., Journel, H., Lazaro, L., Andoni Urtizberea, J., Moerman, A., Faivre, L., Eymard, B., Maincent, K., Gherardi, R., Chaigne, D., Ben Yaou, R., Leturcq, F., Chelly, J., Desguerre, I. :
Genetic and clinical specificity of 26 symptomatic carriers for dystrophinopathies at pediatric age Eur J Hum Genet, 2013 ; 21 (8) : 855-863
Genetic and clinical specificity of 26 symptomatic carriers for dystrophinopathies at pediatric age Eur J Hum Genet, 2013 ; 21 (8) : 855-863
Gallais, B., Gargiulo, M., Montreuil, M., Eymard, B. :
La fatigue dans la dystrophie myotonique de Steinert : revue de la littérature et voies de recherche Annales médico-psychologiques, 2013 ; 171 (2) : 83-88
La fatigue dans la dystrophie myotonique de Steinert : revue de la littérature et voies de recherche Annales médico-psychologiques, 2013 ; 171 (2) : 83-88
Justo, D., Charles, P., Daunizeau, J., Delmaire, C., Gargiulo, M., Hahn-Barma, V., Naccache, L., Durr, A. :
Is non-recognition of choreic movements in Huntington disease always pathological ? Neuropsychologia, 2013 ; 51 (4) : 748-59
Is non-recognition of choreic movements in Huntington disease always pathological ? Neuropsychologia, 2013 ; 51 (4) : 748-59
Masat, E., Pavani, G., Mingozzi, F. :
Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions Discov Med, 2013 ; 15 (85) : 379-89
Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions Discov Med, 2013 ; 15 (85) : 379-89
Ferreboeuf, M., Mariot, V., Bessieres, B., Vasiljevic, A., Attie-Bitach, T., Collardeau, S., Morere, J., Roche, S., Magdinier, F., Robin-Ducellier, J., Rameau, P., Whalen, S., Desnuelle, C., Sacconi, S., Mouly, V., Butler-Browne, G., Dumonceaux, J. :
DUX4 and DUX4 downstream target genes are expressed in fetal FSHD muscles Hum Mol Genet, 2013 ; 23 (1) : 171-81
DUX4 and DUX4 downstream target genes are expressed in fetal FSHD muscles Hum Mol Genet, 2013 ; 23 (1) : 171-81
McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G., Christophe, O. D., McVey, J. H., Waddington, S., Nienhuis, A. W., Gray, J. T., Fagone, P., Mingozzi, F., Zhou, S. Z., High, K. A., Cancio, M., Ng, C. Y., Zhou, J., Morton, C. L., Davidoff, A. M., Nathwani, A. C. :
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant Blood, 2013 ; 121 (17) : 3335-44
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant Blood, 2013 ; 121 (17) : 3335-44
Cufi, P., Dragin, N., Weiss, J. M., Martinez-Martinez, P., De Baets, M. H., Roussin, R., Fadel, E., Berrih-Aknin, S., Le Panse, R. :
Implication of double-stranded RNA signaling in the etiology of autoimmune myasthenia gravis Ann Neurol, 2013 ; 73 (2) : 281-93
Implication of double-stranded RNA signaling in the etiology of autoimmune myasthenia gravis Ann Neurol, 2013 ; 73 (2) : 281-93
