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
Publications (1184)
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
Bencze, M., Negroni, E., Vallese, D., Yacoub-Youssef, H., Chaouch, S., Wolff, A., Aamiri, A., Di Santo, J. P., Chazaud, B., Butler-Browne, G., Savino, W., Mouly, V., Riederer, I. :
Proinflammatory Macrophages Enhance the Regenerative Capacity of Human Myoblasts by Modifying Their Kinetics of Proliferation and Differentiation Mol Ther, 2012 ; 20 (11) : 2168-2179
Proinflammatory Macrophages Enhance the Regenerative Capacity of Human Myoblasts by Modifying Their Kinetics of Proliferation and Differentiation Mol Ther, 2012 ; 20 (11) : 2168-2179
Friedrich, F. W., Wilding, B. R., Reischmann, S., Crocini, C., Lang, P., Charron, P., Muller, O. J., McGrath, M. J., Vollert, I., Hansen, A., Linke, W. A., Hengstenberg, C., Bonne, G., Morner, S., Wichter, T., Madeira, H., Arbustini, E., Eschenhagen, T., Mitchell, C. A., Isnard, R., Carrier, L. :
Evidence for FHL1 as a novel disease gene for isolated hypertrophic cardiomyopathy Hum Mol Genet, 2012 ; 21 (14) : 3237-3254
Evidence for FHL1 as a novel disease gene for isolated hypertrophic cardiomyopathy Hum Mol Genet, 2012 ; 21 (14) : 3237-3254
Lillis, S., Abbs, S., Ferreiro, A., Muntoni, F., Jungbluth, H. :
Clinical utility gene card for: Multi-minicore disease Eur J Hum Genet, 2012 ; 20 (2) :
Clinical utility gene card for: Multi-minicore disease Eur J Hum Genet, 2012 ; 20 (2) :
Stanciu, R., Guiguet, M., Musset, L., Touitou, D., Beigelman, C., Rigolet, A., Costedoat-Chalumeau, N., Allenbach, Y., Hervier, B., Dubourg, O., Maisonobe, T., Charuel, J. L., Behin, A., Herson, S., Amoura, Z., Grenier, P., Benveniste, O. :
Antisynthetase Syndrome with Anti-Jo1 Antibodies in 48 Patients: Pulmonary Involvement Predicts Disease-modifying Antirheumatic Drug Use J Rheumatol, 2012 ; 39 (9) : 1835-9
Antisynthetase Syndrome with Anti-Jo1 Antibodies in 48 Patients: Pulmonary Involvement Predicts Disease-modifying Antirheumatic Drug Use J Rheumatol, 2012 ; 39 (9) : 1835-9
Borrego-Pinto, J., Jegou, T., Osorio, D. S., Aurade, F., Gorjanacz, M., Koch, B., Mattaj, I. W., Gomes, E. R. :
Samp1 is a component of TAN lines and is required for nuclear movement J Cell Sci, 2012 ; 125 (Pt 5) : 1099-10105
Samp1 is a component of TAN lines and is required for nuclear movement J Cell Sci, 2012 ; 125 (Pt 5) : 1099-10105
Hadj-Said, W., Bangratz, M., Vignaud, A., Chatonnet, A., Butler-Browne, G., Nicole, S., Agbulut, O., Ferry, A. :
Effect of locomotor training on muscle performance in the context of nerve-muscle communication dysfunction Muscle Nerve, 2012 ; 45 (4) : 567-577
Effect of locomotor training on muscle performance in the context of nerve-muscle communication dysfunction Muscle Nerve, 2012 ; 45 (4) : 567-577
Mochel, F., Schiffmann, R., Steenweg, M. E., Akman, H. O., Wallace, M., Sedel, F., Laforet, P., Levy, R., Powers, J. M., Demeret, S., Maisonobe, T., Froissart, R., Da Nobrega, B. B., Fogel, B. L., Natowicz, M. R., Lubetzki, C., Durr, A., Brice, A., Rosenmann, H., Barash, V., Kakhlon, O., Gomori, J. M., van der Knaap, M. S., Lossos, A. :
Adult polyglucosan body disease: Natural History and Key Magnetic Resonance Imaging Findings Ann Neurol, 2012 ; 72 (3) : 433-441
Adult polyglucosan body disease: Natural History and Key Magnetic Resonance Imaging Findings Ann Neurol, 2012 ; 72 (3) : 433-441
Wahbi, K., Behin, A., Charron, P., Dunand, M., Richard, P., Meune, C., Vicart, P., Laforet, P., Stojkovic, T., Becane, H. M., Kuntzer, T., Duboc, D. :
High cardiovascular morbidity and mortality in myofibrillar myopathies due to DES gene mutations: a 10-year longitudinal study Neuromuscul Disord, 2012 ; 22 (3) : 211-218
High cardiovascular morbidity and mortality in myofibrillar myopathies due to DES gene mutations: a 10-year longitudinal study Neuromuscul Disord, 2012 ; 22 (3) : 211-218
