Simmons, 2016 - Google Patents
Treatment of DMD 5'Mutations through Two Different Exon 2 Skipping Strategies: rAAV9. U7snRNA Mediated Skipping and Antisense Morpholino OligomersSimmons, 2016
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- 2268253549478914005
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- Simmons T
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Duchenne muscular dystrophy (DMD) is an X-linked progressive disease characterized by loss of dystrophin protein as a result of truncating mutations in the DMD gene. Initial exon- skipping therapies have sought to treat deletion mutations that abolish an open reading …
- 230000035772 mutation 0 title abstract description 69
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