Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
Duchenne muscular dystrophy is a severe muscle-wasting disease caused by mutations in the dystrophin gene that ablate functional protein expression. Although exonic deletions are the most common Duchenne muscular dystrophy lesion, duplications account for 10–15% of reported disease-causing mutations...
| Main Authors: | , , , , |
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| Format: | Online |
| Language: | English |
| Published: |
Nature Publishing Group
2014
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3982197/ |