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Functional repair of a mutant chloride channel using a trans-splicing ribozyme
RNA repair has been proposed as a novel gene-based therapeutic strategy. Modified Tetrahymena group I intron ribozymes have been used to mediate trans-splicing of therapeutically relevant RNA transcripts, but the efficiency of the ribozyme-mediated RNA repair process has not been determined precisel...
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| Autori principali: | , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society for Clinical Investigation
2002
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC151654/ https://ncbi.nlm.nih.gov/pubmed/12488428 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI200216481 |
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