Evidence map›Paper›PMID 26655495›Full record

ArticleNature biotechnology2016

Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay.

Tomoki T Nomakuchi, Frank Rigo, Isabel Aznarez, Adrian R Krainer

Abstract read
In one paragraph

Article in Nature biotechnology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 66 papers.

0numbers the graph read from it
0cells of the map it votes in
66citing papers in PubMed
–field-weighted citation impact
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

66 citing papers in PubMed.

  1. TargetingGenes & development · 2026
    Article
  2. Antisense Oligonucleotide Therapeutics Targeting Age-Related Diseases.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
    Review
  3. A Translational Roadmap for Neurological Nonsense Mutation Disorders.International journal of molecular sciences · 2026
    Review
  4. Article
  5. Review
  6. Review
  7. Article
  8. Article
  9. Review
  10. Review
  11. Chemical Modifications in Nucleic Acid Therapeutics.Methods in molecular biology (Clifton, N.J.) · 2025
    Review
  12. Suppressor tRNA in gene therapy.Science China. Life sciences · 2024
    Review
  13. Systematic deletion of symmetricalNAR molecular medicine · 2024
    Article
  14. Article
  15. RNA Sequencing in Disease Diagnosis.Annual review of genomics and human genetics · 2024
    Review
  16. Review
  17. Article
  18. Article
  19. Article
  20. Article

6 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

4 authors.

Tomoki T NomakuchiCold Spring Harbor Laboratory, Cold Spring Harbor, New York, USA.
Frank RigoIsis Pharmaceuticals, Carslbad, California, USA.
Isabel AznarezCold Spring Harbor Laboratory, Cold Spring Harbor, New York, USA.
Adrian R KrainerCold Spring Harbor Laboratory, Cold Spring Harbor, New York, USA.

Funding

Single-Cell Biology Shared ResourceP30CA045508 · NCI · COLD SPRING HARBOR LABORATORY · PI John Edward Moses · 1987 to 2026
$118.9M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM008444 · NIGMS · STATE UNIVERSITY NEW YORK STONY BROOK · PI FROHMAN, MICHAEL A. · 1992 to 2024
$12.6M
BIOCHEMISTRY OF PREMRNA SPLICINGR01GM042699 · NIGMS · COLD SPRING HARBOR LABORATORY · PI Adrian R Krainer · 1989 to 2026
$8.8M
Biochemistry of Pre-mRNA SplicingR37GM042699 · NIGMS · COLD SPRING HARBOR LABORATORY · PI KRAINER, ADRIAN R · 2012 to 2021
$7.7M
Targeted Inhibition of NMD to Enhance the Efficacy of Readthrough DrugsR21NS081448 · NINDS · COLD SPRING HARBOR LABORATORY · PI KRAINER, ADRIAN R · 2012 to 2013
$509k
Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decayF31NS087747 · NINDS · COLD SPRING HARBOR LABORATORY · PI NOMAKUCHI, TOMOKI T · 2015 to 2015
$27k
NCI NIH HHS 5P30CA045508NCI NIH HHS P30 CA045508NIGMS NIH HHS R01 GM042699NIGMS NIH HHS R37 GM042699NIGMS NIH HHS R37-GM042699NIGMS NIH HHS T32GM008444NINDS NIH HHS F31NS087747NINDS NIH HHS R21 NS081448NINDS NIH HHS R21-NS081448
6 · The paper itself

Abstract

Nonsense-mediated mRNA decay (NMD) is a cellular quality-control mechanism that is thought to exacerbate the phenotype of certain pathogenic nonsense mutations by preventing the expression of semi-functional proteins. NMD also limits the efficacy of read-through compound (RTC)-based therapies. Here, we report a gene-specific method of NMD inhibition using antisense oligonucleotides (ASOs) and combine this approach with an RTC to effectively restore the expression of full-length protein from a nonsense-mutant allele.

Indexed as

Cell Line, TumorGene Expression RegulationGenetic TherapyHumansNonsense Mediated mRNA DecayOligonucleotides, AntisenseReproducibility of ResultsOligonucleotides, Antisense

Identifiers

PMID26655495
PMCPMC4744113

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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.