RNA-targeted splice-correction therapy for neuromuscular disease.
Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing is engineered to yield genetic correction, is a promising novel therapy for genetic diseases of muscle and nerve-the prototypical example being Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most...
| Main Authors: | , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2010
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