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Human Molecular Genetics|February 8, 2017
Exploiting the CRISPR/Cas9 system to study alternative splicing in vivo: application to titinKarine Charton, Laurence Suel, Sara F Henriques, et al.
Scientific Reports|September 11, 2024
Correction of exon 2, exon 2-9 and exons 8-9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 systemJuliette Lemoine, Auriane Dubois, Alan Dorval, et al.
Molecular Therapy. Nucleic Acids|June 19, 2017
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 SystemAnnalisa Lattanzi, Stephanie Duguez, Arianna Moiani, et al.
Human Gene Therapy|September 21, 2012
Antisense-induced messenger depletion corrects a COL6A2 dominant mutation in Ullrich myopathyFrancesca Gualandi, Elisa Manzati, Patrizia Sabatelli, et al.
Disease Models & Mechanisms|January 8, 2026
A new dystrophin deficient rat model mirroring exon skipping in patients with DMD exon 45 deletionsTao Wang, Cynthia Daoud, Auriane Dubois, et al.
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