ReviewInternational journal of molecular medicine2014
Therapeutic suppression of premature termination codons: mechanisms and clinical considerations (review).
Review in International journal of molecular medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
22 citing papers in PubMed, 43 citations in OpenAlex.
- Single-strand deaminase-assisted editing for functional RNA manipulation.Nature biotechnology · 2026Article
- Peer victimization in adolescence alters gene expression and cytokine profiles during transition to adulthood.Brain, behavior, & immunity - health · 2025Article
- Applications of genetic code expansion technology in eukaryotes.Protein & cell · 2024Review
- Targeted pseudouridylation: An approach for suppressing nonsense mutations in disease genes.Molecular cell · 2023Article
- Mini-dCas13X-mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy.The Journal of clinical investigation · 2023Article
- Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives.Frontiers in neuroscience · 2023Review
- Noncanonical Amino Acid Incorporation in Mice.Methods in molecular biology (Clifton, N.J.) · 2023Article
- A systematic dissection of determinants and consequences of snoRNA-guided pseudouridylation of human mRNA.Nucleic acids research · 2022Article
- Restoration of dystrophin expression in mice by suppressing a nonsense mutation through the incorporation of unnatural amino acids.Nature biomedical engineering · 2022Article
- Ataluren-Promising Therapeutic Premature Termination Codon Readthrough Frontrunner.Pharmaceuticals (Basel, Switzerland) · 2021Review
- Therapeutic promise of engineered nonsense suppressor tRNAs.Wiley interdisciplinary reviews. RNA · 2021Review
- MMADHC premature termination codons in the pathogenesis of cobalamin D disorder: Potential of translational readthrough reconstitution.Molecular genetics and metabolism reports · 2021Article
- Pseudouridine-mediated stop codon readthrough inRNA (New York, N.Y.) · 2020Article
- Suppression of Nonsense Mutations by New Emerging Technologies.International journal of molecular sciences · 2020Review
- Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides.Human molecular genetics · 2020Article
- Strategies against Nonsense: Oxadiazoles as Translational Readthrough-Inducing Drugs (TRIDs).International journal of molecular sciences · 2019Review
- Therapeutic developments for Duchenne muscular dystrophy.Nature reviews. Neurology · 2019Review
- Translating the epitranscriptome.Wiley interdisciplinary reviews. RNA · 2017Review
- Hydroxylation and translational adaptation to stress: some answers lie beyond the STOP codon.Cellular and molecular life sciences : CMLS · 2016Review
- Gene-targeting pharmaceuticals for single-gene disorders.Human molecular genetics · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
An estimated one-third of genetic disorders are the result of mutations that generate premature termination codons (PTCs) within protein coding genes. These disorders are phenotypically diverse and consist of diseases that affect both young and old individuals. Various small molecules have been identified that are capable of modulating the efficiency of translation termination, including select antibiotics of the aminoglycoside family and multiple novel synthetic molecules, including PTC124. Several of these agents have proved their effectiveness at promoting nonsense suppression in preclinical animal models, as well as in clinical trials. In addition, it has recently been shown that box H/ACA RNA-guided peudouridylation, when directed to modify PTCs, can also promote nonsense suppression. In this review, we summarize our current understanding of eukaryotic translation termination and discuss various methods for promoting the read-through of disease-causing PTCs, as well as the current obstacles that stand in the way of using the discussed agents broadly in clinical practice.
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Identifiers
What OpenQuestion holds
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.