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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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Hlavní autoři: Rogers, Christopher S., Vanoye, Carlos G., Sullenger, Bruce A., George, Alfred L.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society for Clinical Investigation 2002
Témata:
On-line přístup: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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