By-passing the nonsense mutation in the 4^CV mouse model of muscular dystrophy by induced exon skipping

• Main Authors: Chalermchai Mitrpant, Sue Fletcher, Patrick L. Iversen, Steve D. Wilton
• Other Authors: University of Western Australia
• Format: Article
• Published: 2018
• Subjects: Biochemistry, Genetics and Molecular Biology; Medicine; Pharmacology, Toxicology and Pharmaceutics
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/27212
• Institution: Mahidol University

Background: Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by protein-truncating mutations in the dystrophin gene. Absence of functional dystrophin renders muscle fibres more vulnerable to damage and necrosis. We report antisense oligomer (AO) induced exon skipping in...