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...
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| Main Authors: | , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2014-01-01T00:00:00Z.
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