Evidence map›Paper›PMID 39667907›Full record

ReviewRNA (New York, N.Y.)2025

Exploring the therapeutic potential of modulating nonsense-mediated mRNA decay.

Mary McMahon, Lynne E Maquat

Erratum issuedAbstract readReview
In one paragraph

Review in RNA (New York, N.Y.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing 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

10 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Article
  5. Article
  6. Review
  7. Review
  8. Article
  9. Review
  10. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

2 authors.

Mary McMahonReviR Therapeutics, Brisbane, California 94005, USA mary.mcmahon@revirtx.com lynne_maquat@urmc.rochester.edu.
Lynne E MaquatDepartment of Biochemistry and Biophysics, School of Medicine and Dentistry, University of Rochester, Rochester, New York 14642, USA mary.mcmahon@revirtx.com lynne_maquat@urmc.rochester.edu.

Funding

Nonsense-mediated mRNA decay and beyondR35GM149268 · NIGMS · UNIVERSITY OF ROCHESTER · PI Lynne E Maquat · 2023 to 2026
$3.1M
NIGMS NIH HHS R35 GM149268
6 · The paper itself

Abstract

Discovered more than four decades ago, nonsense-mediated mRNA decay (NMD) plays a fundamental role in the regulation of gene expression and is a major contributor to numerous diseases. With advanced technologies, several novel approaches aim to directly circumvent the effects of disease-causing frameshift and nonsense mutations. Additional therapeutics aim to globally dampen the NMD pathway in diseases associated with pathway hyperactivation, one example being Fragile X syndrome. In other cases, therapeutics have been designed to hijack or inhibit the cellular NMD machinery to either activate or obviate transcript-specific NMD by modulating pre-mRNA splicing. Here, we discuss promising approaches employed to regulate NMD for therapeutic purposes and highlight potential challenges in future clinical development. We are optimistic that the future of developing target-specific and global modulators of NMD (inhibitors as well as activators) is bright and will revolutionize the treatment of many genetic disorders, especially those with high unmet medical need.

Indexed as

Nonsense Mediated mRNA DecayRNA, MessengerAnimalsCodon, NonsenseFragile X SyndromeGene Expression RegulationHumansRNA SplicingCodon, NonsenseRNA, Messengergene therapyNMDpre-mRNA splicingsite-directed RNA editingsmall-molecule modulators of splicingsmall-molecule modulators of translation termination

Identifiers

PMID39667907
PMCPMC11874985

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.