ArticleThe Journal of clinical investigation2002
Functional repair of a mutant chloride channel using a trans-splicing ribozyme.
Article in The Journal of clinical investigation, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
25 citing papers in PubMed, 87 citations in OpenAlex.
- Repair of MutatedCancers · 2025Article
- A split ribozyme system for in vivo plant RNA imaging and genetic engineering.Plant biotechnology journal · 2025Article
- Therapeutic applications of trans-splicing.British medical bulletin · 2020Article
- Mutation-Directed Therapeutics for Neurofibromatosis Type I.Molecular therapy. Nucleic acids · 2020Review
- Specific and Efficient Regression of Cancers Harboring KRAS Mutation by Targeted RNA Replacement.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Targeted Regression of Hepatocellular Carcinoma by Cancer-Specific RNA Replacement through MicroRNA Regulation.Scientific reports · 2015Article
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- Effective suppression of dengue virus using a novel group-I intron that induces apoptotic cell death upon infection through conditional expression of the Bax C-terminal domain.Virology journal · 2014Article
- Article
- Article
- Low selection pressure aids the evolution of cooperative ribozyme mutations in cells.The Journal of biological chemistry · 2013Article
- Computational prediction of efficient splice sites for trans-splicing ribozymes.RNA (New York, N.Y.) · 2012Article
- Targeting of highly conserved Dengue virus sequences with anti-Dengue virus trans-splicing group I introns.BMC molecular biology · 2010Article
- The application of ribozymes and DNAzymes in muscle and brain.Molecules (Basel, Switzerland) · 2010Review
- The non-dystrophic myotonias: molecular pathogenesis, diagnosis and treatment.Brain : a journal of neurology · 2010Review
- Statins and fenofibrate affect skeletal muscle chloride conductance in rats by differently impairing ClC-1 channel regulation and expression.British journal of pharmacology · 2009Article
- Validation of tissue-specific promoter-driven tumor-targeting trans-splicing ribozyme system as a multifunctional cancer gene therapy device in vivo.Cancer gene therapy · 2009Article
- Treatment of neuromuscular channelopathies: current concepts and future prospects.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2008Review
- Proximity-dependent and proximity-independent trans-splicing in mammalian cells.RNA (New York, N.Y.) · 2008Article
- Gene therapy progress and prospects: reprograming gene expression by trans-splicing.Gene therapy · 2005Review
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
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 ribozyme that targets the mRNA of a mutant canine skeletal muscle chloride channel (cClC-1) (mutation T268M in ClC-1 causing myotonia congenita) and replaces the mutant-containing 3' portion by trans-splicing the corresponding 4-kb wild-type sequence. Repair efficiency assessed by quantitative RT-PCR was 1.2% +/- 0.1% in a population of treated cells. However, when chloride channel function was examined in single cells, a wide range of electrophysiological activity was observed, with 18% of cells exhibiting significant functional restoration and some cells exhibiting complete rescue of the biophysical phenotype. These results indicate that RNA repair can restore wild-type protein activity and reveal considerable cell-to-cell variability in ribozyme-mediated trans-splicing reaction efficiency.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.