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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

✍ Scribed by Alter, Julia; Lou, Fang; Rabinowitz, Adam; Yin, HaiFang; Rosenfeld, Jeffrey; Wilton, Steve D; Partridge, Terence A; Lu, Qi Long


Book ID
109932801
Publisher
Nature Publishing Group
Year
2006
Tongue
English
Weight
318 KB
Volume
12
Category
Article
ISSN
1078-8956

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## 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