Evidence map›Paper›PMID 37253858›Full record

ReviewNature reviews. Drug discovery2023

Amplifying gene expression with RNA-targeted therapeutics.

Olga Khorkova, Jack Stahl, Aswathy Joji, Claude-Henry Volmar, Claes Wahlestedt

Open access · bronzeAbstract readReview
In one paragraph

Review in Nature reviews. Drug discovery, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 64 papers.

0numbers the graph read from it
0cells of the map it votes in
64citing papers in PubMed
18.6field-weighted citation impact, top 1% 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

64 citing papers in PubMed, 121 citations in OpenAlex.

  1. Review
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  9. Article
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  15. Review
  16. mRNA vaccines for gastrointestinal cancers: a paradigm shift in treatment.Naunyn-Schmiedeberg's archives of pharmacology · 2026
    Review
  17. mActa pharmaceutica Sinica. B · 2025
    Review
  18. Article
  19. RNA chemistry and therapeutics.Nature reviews. Drug discovery · 2025
    Review
  20. Review

4 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

5 authors at 1 institution in 1 country.

Olga KhorkovaOPKO Health, Miami, FL, USA.
Jack StahlCenter for Therapeutic Innovation, University of Miami, Miami, FL, USA.
Aswathy JojiCenter for Therapeutic Innovation, University of Miami, Miami, FL, USA.
Claude-Henry VolmarCenter for Therapeutic Innovation, University of Miami, Miami, FL, USA.
Claes WahlestedtCenter for Therapeutic Innovation, University of Miami, Miami, FL, USA. cwahlestedt@med.miami.edu.ORCID http://orcid.org/0000-0003-4471-5916
University of Miami · US

Funding

Epigenetic modulation of amygdalar circuits that control alcohol compulsivityR01AA029924 · NIAAA · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI Luis Miguel Tuesta, Claes Robert Wahlestedt · 2022 to 2026
$2.1M
The Role of Menopause-Driven DNA Damage and Epigenetic Dysregulation in Alzheimer s DiseaseR01AG079373 · NIA · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI VOLMAR, CLAUDE-HENRY, WAHLESTEDT, CLAES ROBERT · 2022 to 2025
$1.9M
NIAAA NIH HHS R01 AA029924NIA NIH HHS R01 AG079373
6 · The paper itself

Abstract

Many diseases are caused by insufficient expression of mutated genes and would benefit from increased expression of the corresponding protein. However, in drug development, it has been historically easier to develop drugs with inhibitory or antagonistic effects. Protein replacement and gene therapy can achieve the goal of increased protein expression but have limitations. Recent discoveries of the extensive regulatory networks formed by non-coding RNAs offer alternative targets and strategies to amplify the production of a specific protein. In addition to RNA-targeting small molecules, new nucleic acid-based therapeutic modalities that allow highly specific modulation of RNA-based regulatory networks are being developed. Such approaches can directly target the stability of mRNAs or modulate non-coding RNA-mediated regulation of transcription and translation. This Review highlights emerging RNA-targeted therapeutics for gene activation, focusing on opportunities and challenges for translation to the clinic.

Indexed as

RNAGene ExpressionHumansRNA

Identifiers

PMID37253858
PMCPMC10227815
OpenAlexW4378782363

What OpenQuestion holds

Textmetadata
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.