Popplewell, L., Koo, T., Leclerc, X., Duclert, A., Mamchaoui, K., Gouble, A., Mouly, V., Voit, T., Paques, F., Cedrone, F., Isman, O., Yanez-Munoz, R. J., Dickson, G. :
Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in
Hum Gene Ther, 2013 ; 24 (7) : 692-701
Publications (1184)
Le Hir, M., Goyenvalle, A., Peccate, C., Precigout, G., Davies, K. E., Voit, T., Garcia, L., Lorain, S. :
AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy Mol Ther, 2013 ; 21 (8) : 1551-1558
AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy Mol Ther, 2013 ; 21 (8) : 1551-1558
Hourde, C., Joanne, P., Medja, F., Mougenot, N., Jacquet, A., Mouisel, E., Pannerec, A., Hatem, S., Butler-Browne, G., Agbulut, O., Ferry, A. :
Voluntary Physical Activity Protects from Susceptibility to Skeletal Muscle Contraction-Induced Injury But Worsens Heart Function in mdx Mice Am J Pathol, 2013 ; 182 (5) : 1509-1518
Voluntary Physical Activity Protects from Susceptibility to Skeletal Muscle Contraction-Induced Injury But Worsens Heart Function in mdx Mice Am J Pathol, 2013 ; 182 (5) : 1509-1518
Romero, N. B., Clarke, N. F. :
Congenital myopathies Handb Clin Neurol, 2013 ; 113 : 1321-36
Congenital myopathies Handb Clin Neurol, 2013 ; 113 : 1321-36
Servais, L., Baudoin, H., Zehrouni, K., Richard, P., Sternberg, D., Fournier, E., Eymard, B., Stojkovic, T. :
Pregnancy in congenital myasthenic syndrome J Neurol, 2013 ; 260 (3) : 815-819
Pregnancy in congenital myasthenic syndrome J Neurol, 2013 ; 260 (3) : 815-819
Donadille, B., D'Anella, P., Auclair, M., Uhrhammer, N., Sorel, M., Grigorescu, R., Ouzounian, S., Cambonie, G., Boulot, P., Laforet, P., Carbonne, B., Christin-Maitre, S., Bignon, Y. J., Vigouroux, C. :
Partial lipodystrophy with severe insulin resistance and adult progeria Werner syndrome Orphanet J Rare Dis, 2013 ; 8 (1) : 106
Partial lipodystrophy with severe insulin resistance and adult progeria Werner syndrome Orphanet J Rare Dis, 2013 ; 8 (1) : 106
Voermans, N. C., Jungbluth, H., Brusse, E., van Engelen, B. G., Laforet, P. :
Exertional hyperCKemia might be the first manifestation of a genetic disorder Muscle Nerve, 2013 ; 48 (3) : 461-2
Exertional hyperCKemia might be the first manifestation of a genetic disorder Muscle Nerve, 2013 ; 48 (3) : 461-2
McDonald, C. M., Henricson, E. K., Abresch, R. T., Florence, J., Eagle, M., Gappmaier, E., Glanzman, A. M., Spiegel, R., Barth, J., Elfring, G., Reha, A., Peltz, S. W. :
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study Muscle Nerve, 2013 ; 48 (3) : 357-68
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study Muscle Nerve, 2013 ; 48 (3) : 357-68
Hantai, D., Nicole, S., Eymard, B. :
Congenital myasthenic syndromes: an update Curr Opin Neurol, 2013 ; 26 (5) : 561-568
Congenital myasthenic syndromes: an update Curr Opin Neurol, 2013 ; 26 (5) : 561-568
Yiou, R., Hogrel, J. Y., Loche, C. M., Authier, F. J., Lecorvoisier, P., Jouany, P., Roudot-Thoraval, F., Lefaucheur, J. P. :
Periurethral skeletal myofibre implantation in patients with urinary incontinence and intrinsic sphincter deficiency: a phase I clinical trial BJU Int, 2013 ; 111 (7) : 1105-16
Periurethral skeletal myofibre implantation in patients with urinary incontinence and intrinsic sphincter deficiency: a phase I clinical trial BJU Int, 2013 ; 111 (7) : 1105-16
