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Chemical treatment enhances skipping of a mutated exon in the dystrophin gene
Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disease caused by a loss of the dystrophin protein. Control of dystrophin mRNA splicing to convert severe DMD to a milder phenotype is attracting much attention. Here we report a dystrophinopathy patient who has a point mutation in exon 31...
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| Main Authors: | , , , , , , , , |
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| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
Nature Publishing Group
2011
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| Teme: | |
| Online dostop: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3113229/ https://ncbi.nlm.nih.gov/pubmed/21556062 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms1306 |
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