Haurigot, V., Marco, S., Ribera, A., Garcia, M., Ruzo, A., Villacampa, P., Ayuso, E., Anor, S., Andaluz, A., Pineda, M., Garcia-Fructuoso, G., Molas, M., Maggioni, L., Munoz, S., Motas, S., Ruberte, J., Mingozzi, F., Pumarola, M., Bosch, F. :
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
J Clin Invest, 2013 ; (SP) :
Publications (1181)
Mozzetta, C., Consalvi, S., Saccone, V., Tierney, M., Diamantini, A., Mitchell, K. J., Marazzi, G., Borsellino, G., Battistini, L., Sassoon, D., Sacco, A., Puri, P. L. :
Fibroadipogenic progenitors mediate the ability of HDAC inhibitors to promote regeneration in dystrophic muscles of young, but not old Mdx mice EMBO Mol Med, 2013 ; 5 (4) : 626-39
Fibroadipogenic progenitors mediate the ability of HDAC inhibitors to promote regeneration in dystrophic muscles of young, but not old Mdx mice EMBO Mol Med, 2013 ; 5 (4) : 626-39
Cousens, L. P., Najafian, N., Mingozzi, F., Elyaman, W., Mazer, B., Moise, L., Messitt, T. J., Su, Y., Sayegh, M., High, K., Khoury, S. J., Scott, D. W., De Groot, A. S. :
In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity J Clin Immunol, 2013 ; 33 Suppl 1 : S43-9
In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity J Clin Immunol, 2013 ; 33 Suppl 1 : S43-9
Berrih-Aknin, S., Souroujon, M. C. :
Myasthenia gravis: Special issue Autoimmun Rev, 2013 ; 12 (9) : 861-2
Myasthenia gravis: Special issue Autoimmun Rev, 2013 ; 12 (9) : 861-2
Gargiulo, M., Herson, A., Michon, C. C., Hogrel, J. Y., Doppler, V., Laloui, K., Herson, S., Payan, C., Eymard, B., Laforet, P. :
Attitudes and expectations of patients with neuromuscular diseases about their participation in a clinical trial Rev Neurol (Paris), 2013 ; 169 (8-9) : 670-676
Attitudes and expectations of patients with neuromuscular diseases about their participation in a clinical trial Rev Neurol (Paris), 2013 ; 169 (8-9) : 670-676
Stohr, A., Friedrich, F. W., Flenner, F., Geertz, B., Eder, A., Schaaf, S., Hirt, M. N., Uebeler, J., Schlossarek, S., Carrier, L., Hansen, A., Eschenhagen, T. :
Contractile abnormalities and altered drug response in engineered heart tissue from Mybpc3-targeted knock-in mice J Mol Cell Cardiol, 2013 ; 63 : 189-198
Contractile abnormalities and altered drug response in engineered heart tissue from Mybpc3-targeted knock-in mice J Mol Cell Cardiol, 2013 ; 63 : 189-198
Arimura, T., Onoue, K., Takahashi-Tanaka, Y., Ishikawa, T., Kuwahara, M., Setou, M., Shigenobu, S., Yamaguchi, K., Bertrand, A. T., Machida, N., Takayama, K., Fukusato, M., Tanaka, R., Somekawa, S., Nakano, T., Yamane, Y., Kuba, K., Imai, Y., Saito, Y., Bonne, G., Kimura, A. :
Nuclear accumulation of androgen receptor in gender difference of dilated cardiomyopathy due to lamin A/C mutations Cardiovasc Res, 2013 ; 99 (3) : 382-94
Nuclear accumulation of androgen receptor in gender difference of dilated cardiomyopathy due to lamin A/C mutations Cardiovasc Res, 2013 ; 99 (3) : 382-94
Chort, A., Alves, S., Marinello, M., Dufresnois, B., Dornbierer, J. G., Tesson, C., Latouche, M., Baker, D. P., Barkats, M., El Hachimi, K. H., Ruberg, M., Janer, A., Stevanin, G., Brice, A., Sittler, A. :
Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice Brain, 2013 ; 136 (Pt 6) : 1732-1745
Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice Brain, 2013 ; 136 (Pt 6) : 1732-1745
Finsterer, J., Stollberger, C., Wahbi, K. :
Cardiomyopathy in neurological disorders Cardiovasc Pathol, 2013 ; 22 (5) : 389-400
Cardiomyopathy in neurological disorders Cardiovasc Pathol, 2013 ; 22 (5) : 389-400
Mayhew, A., Mazzone, E. S., Eagle, M., Duong, T., Ash, M., Decostre, V., Vandenhauwe, M., Klingels, K., Florence, J., Main, M., Bianco, F., Henrikson, E., Servais, L., Campion, G., Vroom, E., Ricotti, V., Goemans, N., McDonald, C., Mercuri, E. :
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy Dev Med Child Neurol, 2013 ; 55 (11) : 1038-45
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy Dev Med Child Neurol, 2013 ; 55 (11) : 1038-45
