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Article

Functional repair of a mutant chloride channel using a trans-splicing ribozyme.

The Journal of clinical investigation - 1 Dec 2002

Rogers Christopher S, Vanoye Carlos G, Sullenger Bruce A, George Alfred L

Abstract excerpt

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 precisely and subsequent restoration of protein function has been demonstrated only by indirect means. We engineered a...

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Identifiers

PMID: 12488428
DOI: 10.1172/JCI16481
PMCID: PMC151654

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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