Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by mutations in the dystrophin gene. In most cases, the open-reading frame is disrupted which results in the absence of a functional protein. Antisense-mediated exon skipping is one of the most promising approaches for the t...
| Main Authors: | , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2011
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