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Splicing modulation therapy in the treatment of genetic diseases
Antisense-mediated splicing modulation is a tool that can be exploited in several ways to provide a potential therapy for rare genetic diseases. This approach is currently being tested in clinical trials for Duchenne muscular dystrophy and spinal muscular atrophy. The present review outlines the ver...
Gorde:
| Argitaratua izan da: | Appl Clin Genet |
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| Egile Nagusiak: | , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
Dove Medical Press
2014
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4259397/ https://ncbi.nlm.nih.gov/pubmed/25506237 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TACG.S71506 |
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