Evidence map›Paper›PMID 21724467›Full record

ReviewTrends in molecular medicine2011

Myotonic dystrophy mouse models: towards rational therapy development.

Mário Gomes-Pereira, Thomas A Cooper, Geneviève Gourdon

Abstract readReview
In one paragraph

Review in Trends in molecular medicine, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
59citing papers in PubMed, 1 pooled it
3.8field-weighted citation impact, top 6% 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

59 citing papers in PubMed, 1 synthesis or guideline pooled it, 94 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Oleic Acid Levels in HSAInternational journal of molecular sciences · 2026
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  10. Myotonic dystrophies: an update on clinical features, molecular mechanisms, management, and gene therapy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Review
  11. Article
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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

3 authors at 3 institutions in 2 countries.

Mário Gomes-PereiraInserm U781, Université Paris Descartes, Faculté de Medicine Necker Enfants Malades, Paris, France. mario.pereira@inserm.fr
Thomas A Cooper
Geneviève Gourdon
Baylor College of Medicine · USInserm · FRUniversité Paris Cité · FR

Funding

MOLECULAR PATHOGENESIS OF MYOTONIC DYSTROPHYR01AR045653 · NIAMS · BAYLOR COLLEGE OF MEDICINE · PI COOPER, THOMAS A · 1999 to 2018
$8.3M
TROPONIN T ALTERNATIVE SPLICING IN EMBRYONIC HEARTR01HL045565 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI COOPER, THOMAS A · 1996 to 2018
$5.5M
Transcriptome processing networks in skeletal muscle: mechanisms and functionsR01AR060733 · NIAMS · BAYLOR COLLEGE OF MEDICINE · PI COOPER, THOMAS A · 2011 to 2023
$4.0M
NHLBI NIH HHS R01 HL045565NIAMS NIH HHS R01 AR045653NIAMS NIH HHS R01 AR060733
6 · The paper itself

Abstract

DNA repeat expansions can result in the production of toxic RNA. RNA toxicity has been best characterised in the context of myotonic dystrophy. Nearly 20 mouse models have contributed significant and complementary insights into specific aspects of this novel disease mechanism. These models provide a unique resource to test pharmacological, anti-sense, and gene-therapy therapeutic strategies that target specific events of the pathobiological cascade. Further proof-of-principle concept studies and preclinical experiments require critical and thorough analysis of the multiple myotonic dystrophy transgenic lines available. This review provides in-depth assessment of the molecular and phenotypic features of these models and their contribution towards the dissection of disease mechanisms, and compares them with the human condition. More importantly, it provides critical assessment of their suitability and limitations for preclinical testing of emerging therapeutic strategies.

Indexed as

Disease Models, AnimalAlternative SplicingAnimalsCCAAT-Enhancer-Binding Protein-deltaDNA-Binding ProteinsDNA Repeat ExpansionExonsGenetic TherapyHumansMiceMice, TransgenicMyotonic DystrophyRNARNA-Binding ProteinsCCAAT-Enhancer-Binding Protein-deltaCebpd protein, mouseDNA-Binding ProteinsMbnl1 protein, mouseRNARNA-Binding Proteins

Identifiers

PMID21724467
PMCPMC3881009
OpenAlexW1976127874

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.