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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
(2006)
For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of ...
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
(2003)
As a target for gene therapy, Duchenne muscular dystrophy (DMD) presents many obstacles but also an unparalleled prospect for correction by alternative splicing. The majority of mutations in the dystrophin gene occur in ...