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Journal of Neuromuscular Diseases|November 19, 2016
Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' CellsFlorian Barthélémy, Cédric Blouin, Nicolas Wein, et al.
Human Mutation|January 4, 2012
UMD-DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin geneGaelle Blandin, Christophe Beroud, Veronique Labelle, et al.
Human Gene Therapy|January 7, 2021
Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce <i>DMD</i> Exon 2 SkippingLiubov V Gushchina, Emma C Frair, Natalie Rohan, et al.
Frontiers in Cell and Developmental Biology|July 3, 2023
Promising AAV.U7snRNAs vectors targeting <i>DMPK</i> improve DM1 hallmarks in patient-derived cell linesCamila F Almeida, Florence Robriquet, Tatyana A Vetter, et al.
Skeletal Muscle|November 17, 2015
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cellsEmmanuelle Massouridès, Jérôme Polentes, Philippe-Emmanuel Mangeot, et al.
Science Translational Medicine|September 24, 2010
A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathyMartin Krahn, Nicolas Wein, Marc Bartoli, et al.
Molecular Therapy. Methods & Clinical Development|August 11, 2022
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouseNicolas Wein, Tatyana A Vetter, Adeline Vulin, et al.
Molecular Therapy. Nucleic Acids|November 24, 2022
Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophyLiubov V Gushchina, Tatyana A Vetter, Emma C Frair, et al.
Human Mutation|December 5, 2013
The ZZ domain of dystrophin in DMD: making sense of missense mutationsAdeline Vulin, Nicolas Wein, Dana M Strandjord, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 23, 2023
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primatesSamantha Powers, Shibi Likhite, Kamal K Gadalla, et al.
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