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Frontiers in Cell and Developmental Biology|December 10, 2021
The Dysferlin Transcript Containing the Alternative Exon 40a is Essential for Myocyte FunctionsOcéane Ballouhey, Sébastien Courrier, Virginie Kergourlay, et al.Human Mutation|October 15, 2014
Identification of splicing defects caused by mutations in the dysferlin geneVirginie Kergourlay, Ghadi Raï, Gaëlle Blandin, et al.Journal of Neuromuscular Diseases|November 19, 2016
Novel Pathogenic Variants in a French Cohort Widen the Mutational Spectrum of GNE MyopathyMathieu Cerino, Svetlana Gorokhova, Anthony Béhin, et al.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.Annals of Clinical and Translational Neurology|April 26, 2019
Correction of pseudoexon splicing caused by a novel intronic dysferlin mutationJanice A Dominov, Özgün Uyan, Diane McKenna-Yasek, et al.Scientific Reports|March 8, 2025
Establishing a GMP-compliant manufacturing process and phase-appropriate analytics for early development of a FiCAR T-cell product with a novel CAR spacerAnnu Luostarinen, Arja Vuorela, Erja Kerkelä, et al.Pageof 1