Evidence map›Paper›PMID 32935689›Full record

ReviewChemical Society reviews2020

Design of small molecules targeting RNA structure from sequence.

Andrei Ursu, Jessica L Childs-Disney, Ryan J Andrews, Collin A O'Leary, Samantha M Meyer, Alicia J Angelbello, Walter N Moss, Matthew D Disney

Open access · greenAbstract readReview
In one paragraph

Review in Chemical Society reviews, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.

0numbers the graph read from it
0cells of the map it votes in
36citing papers in PubMed
3.3field-weighted citation impact, top 7% 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

36 citing papers in PubMed, 71 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  4. Review
  5. Review
  6. RNA Splicing as a Therapeutic Target in Cancer.Annual review of pharmacology and toxicology · 2026
    Review
  7. Article
  8. Article
  9. [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
  10. Article
  11. Article
  12. Article
  13. Review
  14. Review
  15. Article
  16. Article
  17. Review
  18. Small molecule approaches to targeting RNA.Nature reviews. Chemistry · 2024
    Review
  19. NMRRSC medicinal chemistry · 2024
    Article
  20. 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

8 authors at 2 institutions in 2 countries.

Andrei UrsuDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu.
Jessica L Childs-DisneyDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu.
Ryan J AndrewsRoy J. Carver Department of Biochemistry, Biophysics & Molecular Biology, Iowa State University, Ames, Iowa, USA. wmoss@iastate.edu.ORCID http://orcid.org/0000-0003-0275-0019
Collin A O'LearyRoy J. Carver Department of Biochemistry, Biophysics & Molecular Biology, Iowa State University, Ames, Iowa, USA. wmoss@iastate.edu.ORCID http://orcid.org/0000-0002-4246-6277
Samantha M MeyerDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu.
Alicia J AngelbelloDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu.
Walter N MossRoy J. Carver Department of Biochemistry, Biophysics & Molecular Biology, Iowa State University, Ames, Iowa, USA. wmoss@iastate.edu.
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu.ORCID http://orcid.org/0000-0001-8486-1796
Scripps Research Institute · USIowa State University · US

Funding

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
Sequence-based Design of Small Molecules Targeting RNAR01GM097455 · NIGMS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2012 to 2019
$4.6M
Finding, folding and characterizing the functions of disease-relevant RNA structuresR01GM133810 · NIGMS · IOWA STATE UNIVERSITY · PI Walter Moss · 2019 to 2026
$2.8M
Understanding Epstein Barr virus oncogenicity through non-coding RNA structureR00GM112877 · NIGMS · IOWA STATE UNIVERSITY · PI MOSS, WALTER · 2017 to 2019
$747k
Chemical approaches for precise recognition of a toxic RNA repeatF31NS110269 · NINDS · SCRIPPS FLORIDA · PI ANGELBELLO, ALICIA JEANNINE · 2018 to 2020
$64k
NCI NIH HHS R01 CA249180NIGMS NIH HHS R00 GM112877NIGMS NIH HHS R01 GM097455NIGMS NIH HHS R01 GM133810NINDS NIH HHS F31 NS110269NINDS NIH HHS R35 NS116846
6 · The paper itself

Abstract

The design and discovery of small molecule medicines has largely been focused on a small number of druggable protein families. A new paradigm is emerging, however, in which small molecules exert a biological effect by interacting with RNA, both to study human disease biology and provide lead therapeutic modalities. Due to this potential for expanding target pipelines and treating a larger number of human diseases, robust platforms for the rational design and optimization of small molecules interacting with RNAs (SMIRNAs) are in high demand. This review highlights three major pillars in this area. First, the transcriptome-wide identification and validation of structured RNA elements, or motifs, within disease-causing RNAs directly from sequence is presented. Second, we provide an overview of high-throughput screening approaches to identify SMIRNAs as well as discuss the lead identification strategy, Inforna, which decodes the three-dimensional (3D) conformation of RNA motifs with small molecule binding partners, directly from sequence. An emphasis is placed on target validation methods to study the causality between modulating the RNA motif in vitro and the phenotypic outcome in cells. Third, emergent modalities that convert occupancy-driven mode of action SMIRNAs into event-driven small molecule chemical probes, such as RNA cleavers and degraders, are presented. Finally, the future of the small molecule RNA therapeutics field is discussed, as well as hurdles to overcome to develop potent and selective RNA-centric chemical probes.

Indexed as

AntagomirsDrug DesignHuntington DiseaseMicroRNAsNucleic Acid ConformationRNARNA, ViralSmall Molecule LibrariesAntagomirsMicroRNAsRNARNA, ViralSmall Molecule Libraries

Identifiers

PMID32935689
PMCPMC7707016
OpenAlexW3085874984

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.