T.P.2.08 Dystrophin expression after systemic delivery of morpholino antisense oligonucleotide in mdx mouse: A dose–response analysis

Dystrophin expression in the mdx mouse a

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

✍ Susan Fletcher; Kaite Honeyman; Abbie M. Fall; Penny L. Harding; Russell D. John 📂 Article 📅 2006 🏛 John Wiley and Sons 🌐 English ⚖ 494 KB

## Abstract ## Background Duchenne and Becker muscular dystrophies are allelic disorders arising from mutations in the dystrophin gene. Duchenne muscular dystrophy is characterised by an absence of functional protein, while Becker muscular dystrophy is usually caused by in‐frame deletions allowing