Evidence map›Paper›PMID 40131857›Full record

ReviewBiochemistry2025

The Druggable Transcriptome Project: From Chemical Probes to Precision Medicines.

Matthew D Disney

Abstract readReview
In one paragraph

Review in Biochemistry, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Chemical Biology 2025: Highlights From the Ch/Bi145 Course at Caltech.Chembiochem : a European journal of chemical biology · 2026
    Review
  2. Article
  3. Review
  4. Article
  5. Article
  6. Definition of the components required for selective packaging of coronavirus genomic RNA.Proceedings of the National Academy of Sciences of the United States of America · 2025
    Article
  7. Article
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

1 author.

Matthew D DisneyDepartment of Chemistry, The Herbert Wertheim UF Scripps Institute for Biomedical Innovation and Technology, 130 Scripps Way, Jupiter, Florida 33458, United States.ORCID 0000-0001-8486-1796

Funding

Targeting druggable coronavirus proteinsU19AI171443 · NIAID · SCRIPPS RESEARCH INSTITUTE, THE · PI SUMIT K CHANDA, Arnab Kumar Chatterjee · 2022 to 2026
$101.4M
Translation Accelerator CoreU19AI171421 · NIAID · STANFORD UNIVERSITY · PI JEFFREY S GLENN · 2022 to 2026
$93.4M
Design of Precision Small Molecules Targeting RNA Repeating Transcripts to Manipulate and Study Disease BiologyR35NS116846 · NINDS · UNIVERSITY OF FLORIDA · PI Matthew D Disney · 2020 to 2026
$9.4M
Targeted degradation of RNAs by using small moleculesR01CA249180 · NCI · UNIVERSITY OF FLORIDA · PI Matthew D Disney · 2020 to 2026
$4.8M
NCI NIH HHS R01 CA249180NIAID NIH HHS U19 AI171421NIAID NIH HHS U19 AI171443NINDS NIH HHS R35 NS116846
6 · The paper itself

Abstract

RNA presents abundant opportunities as a drug target, offering significant potential for small molecule medicine development. The transcriptome, comprising both coding and noncoding RNAs, is a rich area for therapeutic innovation, yet challenges persist in targeting RNA with small molecules. RNA structure can be predicted with or without experimental data, but discrepancies with the actual biological structure can impede progress. Prioritizing RNA targets supported by genetic or evolutionary evidence enhances success. Further, small molecules must demonstrate binding to RNA in cells, not solely in vitro, to validate both the target and compound. Effective small molecule binders modulate functional sites that influence RNA biology, as binding to nonfunctional sites requires recruiting effector mechanisms, for example degradation, to achieve therapeutic outcomes. Addressing these challenges is critical to unlocking RNA's vast potential for small molecule medicines, and a strategic framework is proposed to navigate this promising field, with a focus on targeting human RNAs.

Indexed as

Precision MedicineRNASmall Molecule LibrariesTranscriptomeDrug DiscoveryHumansRNASmall Molecule Libraries

Identifiers

PMID40131857
PMCPMC12005196

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.