Evidence map›Paper›PMID 32419578›Full record

ReviewSLAS discovery : advancing life sciences R & D2020

Target-Directed Approaches for Screening Small Molecules against RNA Targets.

Hafeez S Haniff, Laurent Knerr, Jonathan L Chen, Matthew D Disney, Helen L Lightfoot

Open access · bronzeAbstract readReview
In one paragraph

Review in SLAS discovery : advancing life sciences R & D, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
1.8field-weighted citation impact, top 14% 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

18 citing papers in PubMed, 42 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  4. RNA Splicing as a Therapeutic Target in Cancer.Annual review of pharmacology and toxicology · 2026
    Review
  5. Article
  6. Article
  7. Article
  8. [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
  9. Review
  10. Article
  11. Article
  12. Review
  13. Article
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  15. Article
  16. Article
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  18. Review
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 3 institutions in 3 countries.

Hafeez S HaniffDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL, USA.
Laurent KnerrMedicinal Chemistry, Research and Early Development, Cardiovascular, Renal and Metabolism (CVRM), BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden.
Jonathan L ChenDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL, USA.
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL, USA.ORCID 0000-0001-8486-1796
Helen L LightfootFunctional and Mechanistic Safety, R&D, AstraZeneca, Cambridge, UK.ORCID 0000-0002-9292-0087
Scripps Research Institute · USAstraZeneca (Sweden) · SEAstraZeneca (United Kingdom) · GB

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
Sequence-based Design of Small Molecules Targeting RNAR01GM097455 · NIGMS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2012 to 2019
$4.6M
Developing Novel RNA-Based Therapeutics for Parkinson's DiseaseR33NS096032 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI DISNEY, MATTHEW D, MOURADIAN, M. MARAL · 2018 to 2019
$843k
NIGMS NIH HHS R01 GM097455NINDS NIH HHS DP1 NS096898NINDS NIH HHS P01 NS099114NINDS NIH HHS R33 NS096032
6 · The paper itself

Abstract

RNA molecules have a variety of cellular functions that can drive disease pathologies. They are without a doubt one of the most intriguing yet controversial small-molecule drug targets. The ability to widely target RNA with small molecules could be revolutionary, once the right tools, assays, and targets are selected, thereby defining which biomolecules are targetable and what constitutes drug-like small molecules. Indeed, approaches developed over the past 5-10 years have changed the face of small molecule-RNA targeting by addressing historic concerns regarding affinity, selectivity, and structural dynamics. Presently, selective RNA-protein complex stabilizing drugs such as branaplam and risdiplam are in clinical trials for the modulation of

Indexed as

Molecular Targeted TherapyAzo CompoundsDrug DesignHumansMultiprotein ComplexesNeuromuscular AgentsPyrimidinesRNARNA SplicingSmall Molecule LibrariesSurvival of Motor Neuron 2 ProteinAzo CompoundsMultiprotein ComplexesNeuromuscular AgentsPyrimidinesRisdiplamRNASmall Molecule LibrariesSMN2 protein, humanSurvival of Motor Neuron 2 Protein

Identifiers

PMID32419578
PMCPMC7442623
OpenAlexW3027876220

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.