Evidence map›Paper›PMID 37752346›Full record

ArticleGene therapy2024

Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo.

Gabriela Toro Cabrera, Katharina E Meijboom, Abbas Abdallah, Helene Tran, Zachariah Foster, Alexandra Weiss, Nicholas Wightman, Rachel Stock, Tania Gendron, Alisha Gruntman and 4 more

Abstract read
PubMed Publisher
In one paragraph

Article in Gene therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.5field-weighted citation impact, top 10% 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

8 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. Article
  5. Article
  6. Artificial miRNAs and target-mimics as potential tools for crop improvement.Physiology and molecular biology of plants : an international journal of functional plant biology · 2025
    Review
  7. Article
  8. 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

14 authors at 2 institutions in 1 country.

Gabriela Toro Cabrera *Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Katharina E Meijboom *Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.ORCID 0000-0003-0151-2983
Abbas AbdallahDepartment of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Helene TranDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Zachariah FosterDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.ORCID 0000-0002-5813-5202
Alexandra WeissDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Nicholas WightmanDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Rachel StockDepartment of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Tania GendronDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, FL, 32224, USA.
Alisha GruntmanDepartment of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Anthony GiampetruzziDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, FL, 32224, USA.
Robert H BrownDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA. Robert.Brown@umassmed.edu.ORCID 0000-0001-6062-1528
Christian MuellerDepartment of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA. Christian.mueller4@sanofi.com.
University of Massachusetts Chan Medical School · USMayo Clinic in Florida · US

Funding

Silencing C9or72 with rAAV Mediated RNAiR01NS088689 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI BROWN, ROBERT H., MUELLER, CHRISTIAN · 2014 to 2017
$1.6M
NINDS NIH HHS R01 NS088689U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NINDS) NS088689
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects motor neurons, causing progressive muscle weakness and respiratory failure. The presence of an expanded hexanucleotide repeat in chromosome 9 open reading frame 72 (C9ORF72) is the most frequent mutation causing familial ALS and frontotemporal dementia (FTD). To determine if suppressing expression of C9ORF72 gene products can reduce toxicity, we designed a set of artificial microRNAs (amiRNA) targeting the human C9ORF72 gene. Here we report that an AAV9-mediated amiRNA significantly suppresses expression of the C9ORF72 mRNA, protein, and toxic dipeptide repeat proteins generated by the expanded repeat in the brain and spinal cord of C9ORF72 transgenic mice.

Indexed as

Amyotrophic Lateral SclerosisMicroRNAsNeurodegenerative DiseasesAnimalsC9orf72 ProteinDipeptidesDNA Repeat ExpansionHumansMiceMice, TransgenicProteinsC9orf72 ProteinC9orf72 protein, humanDipeptidesMicroRNAsProteins

Identifiers

PMID37752346
OpenAlexW4387055536

What OpenQuestion holds

Textmetadata
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.