-Skipping to new gene therapies for muscular dystrophy-Functional amounts of dystrophin produced by skipping the mutated exon in the mdxdystrophic mouse-Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology-Modular flexibility of dy
-Skipping to new gene therapies for muscular dystrophy
-Functional amounts of dystrophin produced by skipping the mutated exon in the mdxdystrophic mouse
-Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
-Muscling in: Gene therapies for muscular dystrophy target RNA
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