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Frontiers in Cell and Developmental Biology|April 30, 2026
Bridging science and hope: the evolving story of gene therapy for neuromuscular diseasesNicolas Wein, Florian BarthélémyNeuromuscular Disorders : NMD|September 19, 2018
Personalized gene and cell therapy for Duchenne Muscular DystrophyFlorian Barthélémy, Nicolas WeinMethods in Molecular Biology (Clifton, N.J.)|October 1, 2025
Exploring Therapies for Duchenne Muscular Dystrophy Using Transdifferentiated Patient FibroblastsCamila F Almeida, Nicolas WeinPediatric Clinics of North America|May 30, 2015
Genetics and emerging treatments for Duchenne and Becker muscular dystrophyNicolas Wein, Lindsay Alfano, Kevin M FlaniganHuman Gene Therapy|June 18, 2021
U7 snRNA, a Small RNA with a Big Impact in Gene TherapyDaniel Lesman, Yacidzohara Rodriguez, Dhanarajan Rajakumar, et al.Molecular Medicine (Cambridge, Mass.)|May 11, 2011
Translational research and therapeutic perspectives in dysferlinopathiesFlorian Barthélémy, Nicolas Wein, Martin Krahn, et al.Frontiers in Cellular Neuroscience|October 30, 2023
Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatmentJulia Teixeira Oliveira, Christopher Yanick, Nicolas Wein, et al.Journal of Personalized Medicine|December 23, 2022
Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular DisordersCintia Gomez Limia, Megan Baird, Maura Schwartz, et al.Molecular Therapy. Nucleic Acids|January 29, 2021
Designed U7 snRNAs inhibit <i>DUX4</i> expression and improve FSHD-associated outcomes in <i>DUX4</i> overexpressing cells and FSHD patient myotubesAfrooz Rashnonejad, Gholamhossein Amini-Chermahini, Noah K Taylor, et al.Molecular Therapy. Methods & Clinical Development|April 26, 2021
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated <i>DMD</i> exon 2 skippingTabatha R Simmons, Tatyana A Vetter, Nianyuan Huang, et al.Pageof 4