Evidence map›Paper›PMID 33196168›Full record

ArticleACS chemical biology2020

Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD.

Andrei Ursu, Kye Won Wang, Jessica A Bush, Shruti Choudhary, Jonathan L Chen, Jared T Baisden, Yong-Jie Zhang, Tania F Gendron, Leonard Petrucelli, Ilyas Yildirim and 1 more

Abstract read
In one paragraph

Article in ACS chemical biology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing 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

14 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Article
  5. Cryptic intronic transcriptional initiation generates efficient endogenous mRNA templates for C9orf72-associated RAN translation.Proceedings of the National Academy of Sciences of the United States of America · 2025
    Article
  6. Article
  7. Article
  8. Review
  9. Review
  10. Article
  11. Article
  12. Article
  13. Article
  14. Supramolecular Polymorphism of (GInternational journal of molecular sciences · 2021
    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

11 authors.

Andrei UrsuDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Kye Won WangDepartment of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, Florida 33458, United States.
Jessica A BushDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Shruti ChoudharyDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Jonathan L ChenDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.ORCID 0000-0001-7907-9732
Jared T BaisdenDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Yong-Jie ZhangDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, Florida 32224, United States.
Tania F GendronDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, Florida 32224, United States.
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, Florida 32224, United States.
Ilyas YildirimDepartment of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, Florida 33458, United States.ORCID 0000-0001-8357-1922
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.ORCID 0000-0001-8486-1796

Funding

Expanding insights into FTD disease mechanismsR35NS097273 · NINDS · MAYO CLINIC JACKSONVILLE · PI PETRUCELLI, LEONARD · 2017 to 2024
$10.6M
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
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
Bruker Avance III HD 600 MHz NMR spectrometerS10OD021550 · OD · SCRIPPS FLORIDA · PI KOJETIN, DOUGLAS · 2017 to 2017
$600k
NIH HHS S10 OD021550NINDS NIH HHS DP1 NS096898NINDS NIH HHS P01 NS099114NINDS NIH HHS R35 NS097273NINDS NIH HHS R35 NS116846
6 · The paper itself

Abstract

Genetically defined amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), collectively named c9ALS/FTD, are triggered by hexanucleotide GGGGCC repeat expansions [r(G

Indexed as

Amyotrophic Lateral SclerosisC9orf72 ProteinFrontotemporal DementiaG-QuadruplexesHigh-Throughput Screening AssaysHumansMolecular Dynamics SimulationMolecular StructureRNASmall Molecule LibrariesC9orf72 ProteinC9orf72 protein, humanRNASmall Molecule Libraries

Identifiers

PMID33196168
PMCPMC7788565

What OpenQuestion holds

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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.