Rational design of antisense oligomers to induce dystrophin exon skipping

• Main Authors: Chalermchai Mitrpant, Abbie M. Adams, Penny L. Meloni, Francesco Muntoni, Sue Fletcher, Steve D. Wilton
• Other Authors: University of Western Australia
• Format: Article
• Published: 2018
• Subjects: Biochemistry, Genetics and Molecular Biology; Pharmacology, Toxicology and Pharmaceutics
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/27264
• Institution: Mahidol University

Duchenne muscular dystrophy (DMD), one of the most severe neuromuscular disorders of childhood, is caused by the absence of a functional dystrophin. Antisense oligomer (AO) induced exon skipping is being investigated to restore functional dystrophin expression in models of muscular dystrophy and DMD...