Evidence map›Paper›PMID 34677935›Full record

ArticleACS chemical neuroscience2021

A Small Molecule Exploits Hidden Structural Features within the RNA Repeat Expansion That Causes c9ALS/FTD and Rescues Pathological Hallmarks.

Andrei Ursu, Jared T Baisden, Jessica A Bush, Amirhossein Taghavi, Shruti Choudhary, Yong-Jie Zhang, Tania F Gendron, Leonard Petrucelli, Ilyas Yildirim, Matthew D Disney

Abstract read
In one paragraph

Article in ACS chemical neuroscience, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing 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

9 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Article
  5. Article
  6. Article
  7. Review
  8. Article
  9. A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(GProceedings of the National Academy of Sciences of the United States of America · 2022
    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.

Andrei UrsuDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Jared T BaisdenDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.ORCID 0000-0003-4661-7252
Jessica A BushDepartment of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.
Amirhossein TaghaviDepartment of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, Florida 33458, United States.
Shruti ChoudharyDepartment 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 · 2017 to 2021
$6.5M
Bruker Avance III HD 600 MHz NMR spectrometerS10OD021550 · OD · SCRIPPS FLORIDA · PI KOJETIN, DOUGLAS · 2017 to 2017
$600k
NIH HHS S10 OD021550NINDS NIH HHS P01 NS099114NINDS NIH HHS R35 NS097273NINDS NIH HHS R35 NS116846
6 · The paper itself

Abstract

The hexanucleotide repeat expansion GGGGCC [r(G

Indexed as

Amyotrophic Lateral SclerosisFrontotemporal DementiaC9orf72 ProteinDNA Repeat ExpansionHumansRNAC9orf72 ProteinRNAamyotrophic lateral sclerosisbistable RNAfrontotemporal dementiamicrosatellite disordersNMR spectroscopyquinazolinerepeat associate non-ATG (RAN) translationrepeat expansionRNAsmall molecules

Identifiers

PMID34677935
PMCPMC9594102

What Socratic 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 Socratic graph.