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
Publications (1184)
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
Renouil, M., Stojkovic, T., Jacquemont, M. L., Lauret, K., Boue, P., Fourmaintraux, A., Randrianaivo, H., Tallot, M., Mignard, D., Roelens, P., Tabailloux, D., Bernard, R., Cartault, F., Chane-Thien, E., Dubourg, O., Ferrer, X., Sole, G., Fournier, E., Latour, P., Lacour, A., Mignard, C. :
Maladie de Charcot-Marie-Tooth associée au gene de la periaxine (CMT4F) : description clinique, électrophysiologique et génétique de 24 patients Rev Neurol (Paris), 2013 ; 169 (8-9) : 603-612
Maladie de Charcot-Marie-Tooth associée au gene de la periaxine (CMT4F) : description clinique, électrophysiologique et génétique de 24 patients Rev Neurol (Paris), 2013 ; 169 (8-9) : 603-612
Wahbi, K., Algalarrondo, V., Becane, H. M., Fressart, V., Beldjord, C., Azibi, K., Lazarus, A., Berber, N., Radvanyi-Hoffman, H., Stojkovic, T., Behin, A., Laforet, P., Eymard, B., Hatem, S., Duboc, D. :
Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1 Arch Cardiovasc Dis, 2013 ; 106 (12) : 635-43
Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1 Arch Cardiovasc Dis, 2013 ; 106 (12) : 635-43
Hourde, C., Joanne, P., Noirez, P., Agbulut, O., Butler-Browne, G., Ferry, A. :
Protective effect of female gender-related factors on muscle force-generating capacity and fragility in the dystrophic mdx mouse Muscle Nerve, 2013 ; 48 (1) : 68-75
Protective effect of female gender-related factors on muscle force-generating capacity and fragility in the dystrophic mdx mouse Muscle Nerve, 2013 ; 48 (1) : 68-75
Sequeira, V., Wijnker, P. J., Nijenkamp, L. L., Kuster, D. W., Najafi, A., Witjas-Paalberends, R., Regan, J. A., Boontje, N., Ten Cate, F., Germans, T., Carrier, L., Sadayappan, S., van Slegtenhorst, M., Zaremba, R., Foster, D. B., Murphy, A., Poggesi, C., Dos Remedios, C. G., Stienen, G. J., Ho, C. Y., Michels, M., van der Velden, J. :
Perturbed Length-Dependent Activation in Human Hypertrophic Cardiomyopathy with Missense Sarcomeric Gene Mutations Circ Res, 2013 ; 112 (11) : 1491-505
Perturbed Length-Dependent Activation in Human Hypertrophic Cardiomyopathy with Missense Sarcomeric Gene Mutations Circ Res, 2013 ; 112 (11) : 1491-505
Donzelli, S., Fischer, G., King, B. S., Niemann, C., Dumond, J. F., Heeren, J., Wieboldt, H., Baldus, S., Gerloff, C., Eschenhagen, T., Carrier, L., Boger, R. H., Espey, M. G. :
Pharmacological characterization of 1-nitrosocyclohexyl acetate, a long acting nitroxyl donor that shows vasorelaxant and anti-aggregator effect J Pharmacol Exp Ther, 2013 ; 344 (2) : 339-347
Pharmacological characterization of 1-nitrosocyclohexyl acetate, a long acting nitroxyl donor that shows vasorelaxant and anti-aggregator effect J Pharmacol Exp Ther, 2013 ; 344 (2) : 339-347
Michot, C., Mamoune, A., Vamecq, J., Viou, M. T., Hsieh, L. S., Testet, E., Laine, J., Hubert, L., Dessein, A. F., Fontaine, M., Ottolenghi, C., Fouillen, L., Nadra, K., Blanc, E., Bastin, J., Candon, S., Pende, M., Munnich, A., Smahi, A., Djouadi, F., Carman, G. M., Romero, N., de Keyzer, Y., de Lonlay, P. :
Combination of lipid metabolism alterations and their sensitivity to inflammatory cytokines in human lipin-1-deficient myoblasts Biochim Biophys Acta, 2013 ; 1832 (12) : 2103-14
Combination of lipid metabolism alterations and their sensitivity to inflammatory cytokines in human lipin-1-deficient myoblasts Biochim Biophys Acta, 2013 ; 1832 (12) : 2103-14
