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Journal of Neuromuscular Diseases|September 5, 2017
Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide ApproachNicolas Wein, Adeline Vulin, Andrew R Findlay, et al.
Genetic Testing and Molecular Biomarkers|July 15, 2009
Identification of different genomic deletions and one duplication in the dysferlin gene using multiplex ligation-dependent probe amplification and genomic quantitative PCRMartin Krahn, Ana Borges, Claire Navarro, et al.
Human Gene Therapy|June 1, 2021
Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting <i>DMD</i> Exon 2 DuplicationsNicolas Wein, Diane M Dunn, Megan A Waldrop, et al.
Journal of Visualized Experiments : Jove|April 19, 2021
Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular DisordersCamila F Almeida, Emma C Frair, Nianyuan Huang, et al.
Neuromuscular Disorders : NMD|September 15, 2015
The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic developmentAdeline Vulin, Nicolas Wein, Tabatha R Simmons, et al.
Molecular Therapy. Methods & Clinical Development|November 29, 2023
Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse modelLiubov V Gushchina, Adrienne J Bradley, Tatyana A Vetter, et al.
Stem Cell Research|May 7, 2026
Generation of an induced pluripotent stem cell line (NCHi026-A) from a patient with a partial deletion of exon 55 in the DMD geneHui Lin, Stefan Nicolau, Kayla Stevens, et al.
Human Mutation|December 3, 2009
Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skippingNicolas Wein, Aurélie Avril, Marc Bartoli, et al.
Molecular Therapy. Oncology|December 25, 2024
Employing splice-switching oligonucleotides and AAVrh74.U7 snRNA to target insulin receptor splicing and cancer hallmarks in osteosarcomaSafiya Khurshid, Akila S Venkataramany, Matias Montes, et al.
The FEBS Journal|September 14, 2013
Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cellsMirella Meregalli, Claire Navarro, Clementina Sitzia, et al.
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