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Molecular Therapy : the Journal of the American Society of Gene Therapy|May 28, 2014
Blockade of ActRIIB signaling triggers muscle fatigability and metabolic myopathyKarima Relizani, Etienne Mouisel, Benoit Giannesini, et al.
Blood Advances|January 4, 2018
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectorsAmine Meliani, Florence Boisgerault, Zachary Fitzpatrick, et al.
Nature Communications|October 7, 2018
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administrationAmine Meliani, Florence Boisgerault, Romain Hardet, et al.
Human Molecular Genetics|March 9, 2010
Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibresCapucine Trollet, Seyed Yahya Anvar, Andrea Venema, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 4, 2018
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE ExpressionPatrice Vidal, Serena Pagliarani, Pasqualina Colella, et al.
Science Translational Medicine|December 1, 2017
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidaseFrancesco Puzzo, Pasqualina Colella, Maria G Biferi, et al.
The Journal of Cell Biology|December 17, 2009
Muscle inactivation of mTOR causes metabolic and dystrophin defects leading to severe myopathyValérie Risson, Laetitia Mazelin, Mila Roceri, et al.
Science Translational Medicine|January 24, 2014
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathyMartin K Childers, Romain Joubert, Karine Poulard, et al.
Nature Medicine|May 31, 2011
Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophyCharlotte Fugier, Arnaud F Klein, Caroline Hammer, et al.
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