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Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations
RNA trans-splicing has become a versatile tool in the gene therapy of monogenetic diseases. This technique is especially valuable for the correction of mutations in large genes such as COL7A1, which underlie the dystrophic subtype of the skin blistering disease epidermolysis bullosa. Over 800 mutati...
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| Publicado en: | Gene Ther |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Nature Publishing Group
2016
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5097067/ https://ncbi.nlm.nih.gov/pubmed/27434145 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2016.57 |
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