Evidence map›Paper›PMID 31241884›Full record

ArticleACS chemical biology2019

Small Molecule Targeting TDP-43's RNA Recognition Motifs Reduces Locomotor Defects in a

Liberty François-Moutal, Razaz Felemban, David D Scott, Melissa R Sayegh, Victor G Miranda, Samantha Perez-Miller, Rajesh Khanna, Vijay Gokhale, Daniela C Zarnescu, May Khanna

Abstract read
In one paragraph

Article in ACS chemical biology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.

0numbers the graph read from it
0cells of the map it votes in
46citing papers in PubMed
5.6field-weighted citation impact, top 3% 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

46 citing papers in PubMed, 67 citations in OpenAlex.

  1. The cellular landscape of druggable RNA-binding proteins.Nature reviews. Drug discovery · 2026
    Review
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  6. Regulation of RNA-binding proteins by small biomolecules.Nature reviews. Molecular cell biology · 2026
    Review
  7. Review
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  9. Identifying interactions between TDP-43's N-terminal and RNA-binding domains.Protein science : a publication of the Protein Society · 2025
    Article
  10. Review
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  13. Drug repurposing in amyotrophic lateral sclerosis (ALS).Expert opinion on drug discovery · 2025
    Review
  14. Article
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  17. Article
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  20. A Novel Drosophila-based Drug Repurposing Platform Identified Fingolimod As a Potential Therapeutic for TDP-43 Proteinopathy.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023
    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

10 authors at 1 institution in 2 countries.

Liberty François-MoutalDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.
Razaz FelembanDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.
David D ScottDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.
Melissa R SayeghDepartment of Molecular and Cellular Biology , University of Arizona , Tucson , Arizona 85721 , United States.
Victor G MirandaDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.
Samantha Perez-MillerDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.
Rajesh KhannaDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.ORCID 0000-0002-9066-2969
Vijay GokhaleBio5 Institute , University of Arizona , Tucson , Arizona 85721 , United States.
Daniela C ZarnescuDepartment of Molecular and Cellular Biology , University of Arizona , Tucson , Arizona 85721 , United States.
May KhannaDepartment of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.ORCID 0000-0002-9989-6374
University of Arizona · US

Funding

Translation dysregulation in neurodegenerationR01NS091299 · NINDS · UNIVERSITY OF ARIZONA · PI DANIELA C ZARNESCU · 2015 to 2026
$3.4M
NINDS NIH HHS R01 NS091299
6 · The paper itself

Abstract

RNA dysregulation likely contributes to disease pathogenesis of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. A pathological form of the transactive response (TAR) DNA binding protein (TDP-43) binds to RNA in stress granules and forms membraneless, amyloid-like TDP-43 aggregates in the cytoplasm of ALS motor neurons. In this study, we hypothesized that by targeting the RNA recognition motif (RRM) domains of TDP-43 that confer a pathogenic interaction between TDP-43 and RNA, motor neuron toxicity could be reduced.

Indexed as

Amyotrophic Lateral SclerosisAnimalsBase SequenceBinding SitesDNA-Binding ProteinsDrosophila melanogasterDrosophila ProteinsLocomotionMolecular Docking SimulationNeuroprotective AgentsPiperidinesProtein BindingProtein DomainsPyrazinesRNASmall Molecule LibrariesDNA-Binding ProteinsDrosophila ProteinsNeuroprotective AgentsPiperidinesPyrazinesRNASmall Molecule LibrariesTBPH protein, Drosophila

Identifiers

PMID31241884
PMCPMC6911355
OpenAlexW2953872267

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.