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by Schmidt, Manfred and Verhoeyen, Els and Gobbo, Emilie and Divers, Dominique and Oudrhiri, Noufissa and Griscelli, Frank and Bennaceur-Griscelli, Annelise and Klatzmann, David and Anguela, Xavier M and Sharma, Rajiv and Sharma, Rajiv and Li, Hojun and Li, Hojun and Haurigot, Virginia and Haurigot, Virginia and Haurigot, Virginia and Bhagwat, Anand and Bhagwat, Anand and Davidson, Robert and Davidson, Robert and Zhou, Shangzhen and Zhou, Shangzhen and Zhou, Shangzhen and Doyon, Yannick and Gregory, Philip D and Gregory, Philip D and Gregory, Philip D and Holmes, Michael C and Holmes, Michael C and Holmes, Michael C and High, Katherine A and Carbonaro, Denise and Shaw, Kit and Jin, Xiangyang and Geiger, Sabine and Mishra, Suparna and Cooper, Aaron and DeOliveira, Satiro and Sokolic, Rob and Candotti, Fabio and Carmo, Marlene and Arumugam, Paritha and Alonso-Ferrero, Maria and Schambach, Axel and Schambach, Axel and Baum, Christopher and Baum, Christopher and Baum, Christopher and Risma, Kimberly and Malik, Punam and Jordan, Michael and Rivat, Christine and Booth, Claire and Thrasher, Adrian and Whilding, Lynsey and Archibald, Kyra and Oberg, Daniel and Golan, Talia and Hubert, Ayala and Shemi, Amotz and Khvalevsky, Elina Zorde and Gabai-Malka, Racheli and Focht, Gili and Brunschwig, Zivia and Raskin, Stephen and Goldberg, Nahum and Ben-David, Eli and Peretz, Tamar and Eliakim, Rami and Dankur, Alan and Galun and Rachmur, Itzik and Domb, Avi and Kopelman, Yael and Hantz, Yael and Lahav, Mor and Arbel-Alon, Sagit and Dickson, George and Barkats, Martine and Daboussi, Fayza and Silva, Georges and Cedrone, Frederic and Epinat, Jean Charles and Juillerat, Alexandre and Valton, Julien and Montini, Eugenio and Biffi, Alessandra and Biffi, Alessandra and Calabria, Andrea and Calabria, Andrea and Biasco, Luca and Biasco, Luca and Cesani, Martina and Cesani, Martina and Benedicenti, Fabrizio and Benedicenti, Fabrizio and Plati, Tiziana and Leo, Simone and Zanetti, Gianluigi and Aiuti, Alessandro and ...
Human Gene Therapy, ISSN 1043-0342, 10/2012, Volume 23, Issue 10, pp. A1 - A173
Journal Article
Human Molecular Genetics, ISSN 0964-6906, 7/2008, Volume 17, Issue 14, pp. 2132 - 2143
Myotubular myopathy (XLMTM, OMIM 310400) is a severe congenital muscular disease due to mutations in the myotubularin gene (MTM1) and characterized by the... 
MARIE-TOOTH-DISEASE | 3-PHOSPHATE | CENTRONUCLEAR MYOPATHY | PHOSPHATASE | BIOCHEMISTRY & MOLECULAR BIOLOGY | GENETICS & HEREDITY | ADENOASSOCIATED VIRUS VECTORS | DYNAMIN-2 | DUCHENNE MUSCULAR-DYSTROPHY | GENE-THERAPY | AMPHIPHYSIN-2 BIN1 | MUTATIONS | Dependovirus - genetics | Genetic Therapy | Myopathies, Structural, Congenital - physiopathology | Homeostasis | Protein Tyrosine Phosphatases, Non-Receptor - administration & dosage | Male | Muscle, Skeletal - metabolism | Myopathies, Structural, Congenital - genetics | Cell Membrane - genetics | Cell Membrane - chemistry | Muscle, Skeletal - chemistry | Myopathies, Structural, Congenital - therapy | Protein Tyrosine Phosphatases, Non-Receptor - genetics | Cell Membrane - pathology | Female | Cell Membrane - metabolism | Cell Line | Dependovirus - metabolism | Injections, Intramuscular | Genetic Vectors - genetics | Phenotype | Animals | Muscle, Skeletal - physiopathology | Protein Tyrosine Phosphatases, Non-Receptor - metabolism | Mice | Myopathies, Structural, Congenital - metabolism | Muscle, Skeletal - pathology | Protein Tyrosine Phosphatases, Non-Receptor - analysis | Index Medicus | Cell Membrane | Gene Therapy | Protein Tyrosine Phosphatases, Non-Receptor | Dependovirus | Life Sciences | Ethics | Myopathies, Structural, Congenital | Muscle, Skeletal | Genetic Vectors
Journal Article
Journal Article
Nature Communications, ISSN 2041-1723, 07/2017, Volume 8, Issue 1, pp. 16105 - 16105
Journal Article
Molecular Therapy, ISSN 1525-0016, 11/2014, Volume 22, Issue 11, pp. 1923 - 1935
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 02/2013, Volume 8, Issue 2, pp. e55281 - e55281
Journal Article
by Verhoeyen, Els and Erard, Francois and Quesniaux, Valerie J.F and Galy, Anne and Galy, Anne and Boisgerault, Florence and Boisgerault, Florence and Sudres, Muriel and Cire, Séverine and Ryffel, Bernhard and Ferrand, Maxime and Ferrand, Maxime and Darocha, Sylvie and Wilmes, Cyril and Suleman, Muhammad and Galea, Sandra and Gavard, Françoise and Mérillon, Nathalie and Klonjkowski, Bernard and Tartour, Eric and Spaeth, Erika and Andreeff, Michael and Bigey, Pascal and Burgain, Aurore and Escriou, Virginie and Marie, Corinne and Quiviger, Mickael and Schlegel, Anne and Fischer, Alain and Cavazzana-Calvo, Marina and Tuddenham, Edward G.D and Rosales, Cecilia and Macintosh, Jenny and Chowdary, Pratima and Riddell, Anne and Aghighi, Saman and Lilley, Patricia and Yee, Thynn and Griffioen, Anja and Pie, Jun and Harrington, Chris and Glader, Bert and Ng, Catherine Y.C and Kaye, Mark and Zhou, Junfang and Allay, James and Coleman, John and Sleep, Susan and High, Katherine and Mingozzi, Federico and Gray, John T and Reiss, Ulrike M and Nienhuis, Arthur W and Davidoff, Andrew M and Rama, Paolo and Matuska, Stanislav and Paganoni, Giorgio and Spinelli, Alessandra and De Luca, Michele and Thanou, Maya and Holic-Barlet, Nathalie and Frin, Sophie and Galy, Anne and Perea, Javier and Le Gall, Tony and Carmoy, Nathalie and Hyde, Stephen C and Gill, Deborah and Berchel, Mathieu and Fraix, Aurore and Jaffres, Paul-Alain and Lehn, Pierre and Lehn, Pierre and Montier, Tristan and Ferry, Nicolas and Ferry, Nicolas and Ferry, Nicolas and Conchon, Sophie and Conchon, Sophie and Li, Jun and Astord, Stéphanie and Briot-Nivard, Delphine and Wakeling, Erin and Fyfe, John and Costiou, Patrick and Marais, Thibault and Voit, Thomas and Moullier, Philippe and Barkats, Martine and Joussemet, Béatrice and Da Rocha, Sylvie and Corre, Guillaume and Yamagata, Yoshiaki and Sanatine, Peggy and Paldi, Andras and Muscari, Fabrice and Peron, Jean-Marie and Buscail, Louis and Sicard, Flavie and Sicard, Flavie and ...
Human Gene Therapy, ISSN 1043-0342, 06/2011, Volume 22, Issue 6, pp. A-1 - A-29
Journal Article
Human Gene Therapy, ISSN 1043-0342, 06/2016, Volume 27, Issue 9, pp. 712 - 726
Currently, the clinically most advanced strategy to treat Duchenne muscular dystrophy (DMD) is the exon skipping strategy. Whereas antisense... 
Life Sciences | Human health and pathology | Genetics
Journal Article
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 06/2019, Volume 13, pp. 494 - 502
Sarcoglycanopathies are rare autosomic limb girdle muscular dystrophies caused by mutations in one of the genes coding for sarcoglycans. Sarcoglycans form a... 
translational medicine | miRNA biomarker | AAV | γ-sarcoglycanopathy | gene therapy | mechanical stress | limb girdle muscular dystrophy | LGMD2C | MEDICINE, RESEARCH & EXPERIMENTAL | IMAGE | GENE | MUTATION | MIRNAS | SPECTRUM | MUSCLES | Life Sciences | Biotechnology | Immunology | Quantitative Methods | Biochemistry, Molecular Biology | Genetics | Cellular Biology
Journal Article