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: | Kane L Greer, Hanns Lochmüller, Kevin Flanigan, Susan Fletcher, Steve D Wilton |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2014-01-01
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Series: | Molecular Therapy: Nucleic Acids |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2162253116302955 |
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