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Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
Protein-truncating mutations in the dystrophin gene lead to the progressive muscle wasting disorder Duchenne muscular dystrophy, whereas in-frame deletions typically manifest as the milder allelic condition, Becker muscular dystrophy. Antisense oligomer-induced exon skipping can modify dystrophin ge...
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| Main Authors: | , , , , , , , , , |
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| Format: | Artigo |
| Language: | Inglês |
| Published: |
Nature Publishing Group
2012
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3499695/ https://ncbi.nlm.nih.gov/pubmed/23344648 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.40 |
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