Evidence map›Paper›PMID 30778053›Full record

ArticleNature communications2019

Engineered transfer RNAs for suppression of premature termination codons.

John D Lueck, Jae Seok Yoon, Alfredo Perales-Puchalt, Adam L Mackey, Daniel T Infield, Mark A Behlke, Marshall R Pope, David B Weiner, William R Skach, Paul B McCray and 1 more

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 106 papers.

0numbers the graph read from it
0cells of the map it votes in
106citing papers in PubMed
5.1field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

106 citing papers in PubMed, 142 citations in OpenAlex.

  1. RNAViruses · 2026
    Review
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  3. The impact of psuedouridine modification on human tRNA.Biochemical Society transactions · 2026
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  8. The regulation, function and disease relevance of cytoplasmic tRNAs.Nature reviews. Molecular cell biology · 2026
    Review
  9. Self-Assembling Short Peptide Carriers for Gene Delivery.International journal of molecular sciences · 2026
    Review
  10. Article
  11. Article
  12. Article
  13. Article
  14. A Translational Roadmap for Neurological Nonsense Mutation Disorders.International journal of molecular sciences · 2026
    Review
  15. Article
  16. Article
  17. Article
  18. Article
  19. Article
  20. Precision therapeutic tRNA rescue of nonsense mutation R166X inJournal of precision medicine (Amsterdam, Netherlands) · 2025
    Article

46 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 5 institutions in 1 country.

John D LueckDepartment of Physiology and Pharmacology, University of Rochester School of Medicine and Dentistry, Rochester, NY, 14642, USA. john_lueck@urmc.rochester.edu.
Jae Seok YoonCFFT Lab, Cystic Fibrosis Foundation Therapeutics, Lexington, 02421, MA, USA.
Alfredo Perales-PuchaltThe Wistar Institute, Philadelphia, 19104, PA, USA.ORCID http://orcid.org/0000-0003-4904-5050
Adam L MackeyDepartment of Molecular Physiology and Biophysics, Iowa Neuroscience Institute, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.
Daniel T InfieldDepartment of Molecular Physiology and Biophysics, Iowa Neuroscience Institute, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.
Mark A BehlkeIntegrated DNA Technologies Inc., Coralville, IA, 52241, USA.ORCID http://orcid.org/0000-0002-4218-857X
Marshall R PopeDepartment of Molecular Physiology and Biophysics, Iowa Neuroscience Institute, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.
David B WeinerThe Wistar Institute, Philadelphia, 19104, PA, USA.
William R SkachCFFT Lab, Cystic Fibrosis Foundation Therapeutics, Lexington, 02421, MA, USA.
Paul B McCrayStead Family Department of Pediatrics, Pappajohn Biomedical Institute, University of Iowa, Iowa City, IA, 52242, USA.
Christopher A AhernDepartment of Molecular Physiology and Biophysics, Iowa Neuroscience Institute, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA. christopher-ahern@uiowa.edu.ORCID http://orcid.org/0000-0002-7975-2744
University of Iowa · USCystic Fibrosis Foundation · USThe Wistar Institute · USIntegrated DNA Technologies (United States) · USUniversity of Rochester Medical Center · US

Funding

Tumor Microenvironment and MetastasisP30CA010815 · NCI · WISTAR INSTITUTE · PI Luis J Montaner · 1985 to 2026
$75.9M
Vector Core-Core 2P30DK054759 · NIDDK · UNIVERSITY OF IOWA · PI Alejandro Antonio Pezzulo · 1998 to 2026
$30.5M
Chemical Biology of Voltage-Gated Sodium and Potassium ChannelsR01GM106569 · NIGMS · UNIVERSITY OF IOWA · PI AHERN, CHRISTOPHER A · 2013 to 2022
$2.7M
NCI NIH HHS P30 CA010815NIDDK NIH HHS P30 DK054759NIGMS NIH HHS R01 GM106569
6 · The paper itself

Abstract

Premature termination codons (PTCs) are responsible for 10-15% of all inherited disease. PTC suppression during translation offers a promising approach to treat a variety of genetic disorders, yet small molecules that promote PTC read-through have yielded mixed performance in clinical trials. Here we present a high-throughput, cell-based assay to identify anticodon engineered transfer RNAs (ACE-tRNA) which can effectively suppress in-frame PTCs and faithfully encode their cognate amino acid. In total, we identify ACE-tRNA with a high degree of suppression activity targeting the most common human disease-causing nonsense codons. Genome-wide transcriptome ribosome profiling of cells expressing ACE-tRNA at levels which repair PTC indicate that there are limited interactions with translation termination codons. These ACE-tRNAs display high suppression potency in mammalian cells, Xenopus oocytes and mice in vivo, producing PTC repair in multiple genes, including disease causing mutations within cystic fibrosis transmembrane conductance regulator (CFTR).

Indexed as

AnimalsCodon, NonsenseCystic Fibrosis Transmembrane Conductance RegulatorFemaleGene LibraryGenetic EngineeringHEK293 CellsHumansMice, Inbred StrainsOocytesRibosomesRNA, TransferXenopus laevisCFTR protein, humanCodon, NonsenseCystic Fibrosis Transmembrane Conductance RegulatorRNA, Transfer

Identifiers

PMID30778053
PMCPMC6379413
OpenAlexW2949980705

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.