Evidence map›Paper›PMID 32212706›Full record

ArticleJournal of medicinal chemistry2020

How We Think about Targeting RNA with Small Molecules.

Matthew G Costales, Jessica L Childs-Disney, Hafeez S Haniff, Matthew D Disney

Open access · greenAbstract read
In one paragraph

Article in Journal of medicinal chemistry, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 97 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
97citing papers in PubMed, 1 pooled it
8.8field-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

97 citing papers in PubMed, 1 synthesis or guideline pooled it, 156 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. The Role of Long Noncoding RNAs in Modulation of Stress Granules in Cancer.Journal of cellular and molecular medicine · 2026
    Review
  5. Article
  6. Review
  7. Induced proximity-based therapeutic modalities.Nature reviews. Drug discovery · 2026
    Review
  8. Article
  9. Article
  10. Article
  11. Article
  12. Roles of long non-coding RNAs in oesophageal cancer pathogenesis.International journal of experimental pathology · 2025
    Review
  13. Article
  14. [Targeting chimera technology: A new tool for undruggable in breast cancer].Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2025
    Review
  15. Article
  16. Article
  17. Review
  18. Perspectives on Applications ofMolecules (Basel, Switzerland) · 2024
    Review
  19. Article
  20. Article

37 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 at 1 institution in 1 country.

Matthew G CostalesDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Jessica L Childs-DisneyDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Hafeez S HaniffDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.ORCID 0000-0001-8486-1796
Scripps Research Institute · US

Funding

Validation of r(G4C2)exp-binding small molecules in vivo and c9FTD/ALS biomarker developmentP01NS099114 · NINDS · UNIVERSITY OF FLORIDA · PI DISNEY, MATTHEW D, PETRUCELLI, LEONARD · 2017 to 2021
$6.5M
Using a disease-affected cell to synthesize its own drugDP1NS096898 · NINDS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2015 to 2019
$5.0M
Targeted degradation of RNAs by using small moleculesR01CA249180 · NCI · UNIVERSITY OF FLORIDA · PI Matthew D Disney · 2020 to 2026
$4.8M
Sequence-based Design of Small Molecules Targeting RNAR01GM097455 · NIGMS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2012 to 2019
$4.6M
Identifying and Studying RNA Loop-Small Molecule InteractionsR01GM079235 · NIGMS · STATE UNIVERSITY OF NEW YORK AT BUFFALO · PI DISNEY, MATTHEW D · 2008 to 2012
$2.6M
NCI NIH HHS R01 CA249180NIGMS NIH HHS R01 GM079235NIGMS NIH HHS R01 GM097455NINDS NIH HHS DP1 NS096898NINDS NIH HHS P01 NS099114
6 · The paper itself

Abstract

RNA offers nearly unlimited potential as a target for small molecule chemical probes and lead medicines. Many RNAs fold into structures that can be selectively targeted with small molecules. This Perspective discusses molecular recognition of RNA by small molecules and highlights key enabling technologies and properties of bioactive interactions. Sequence-based design of ligands targeting RNA has established rules for affecting RNA targets and provided a potentially general platform for the discovery of bioactive small molecules. The RNA targets that contain preferred small molecule binding sites can be identified from sequence, allowing identification of off-targets and prediction of bioactive interactions by nature of ligand recognition of functional sites. Small molecule targeted degradation of RNA targets (ribonuclease-targeted chimeras, RIBOTACs) and direct cleavage by small molecules have also been developed. These growing technologies suggest that the time is right to provide small molecule chemical probes to target functionally relevant RNAs throughout the human transcriptome.

Indexed as

Drug DesignGenomicsHumansLigandsMicroRNAsNucleic Acid ConformationRibonucleasesRNASmall Molecule LibrariesLigandsMicroRNAsRibonucleasesRNASmall Molecule Libraries

Identifiers

PMID32212706
PMCPMC7486258
OpenAlexW3013781651

What OpenQuestion holds

Textmetadata
LicenceTDM
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.