Evidence map›Paper›PMID 41137396›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026

Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.

Caroline Le Guiner, Gilles Toumaniantz, Thibaut Larcher, Sylvie Marchand, Laurine Buscara, Grégory Cedrone, Cladice Varela Moreira, Amandine Lancelot, Christophe Georger, Aude Lafoux and 11 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Review
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

21 authors.

Caroline Le GuinerNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France. Electronic address: caroline.le-guiner@univ-nantes.fr.
Gilles ToumaniantzNantes Université, CNRS, INSERM, l'Institut du Thorax, 44007 Nantes, France.
Thibaut LarcherINRAE Oniris, UMR 703, PanTher, APEX, 44300 Nantes, France.
Sylvie MarchandGenethon, 91000 Evry, France.
Laurine BuscaraGenethon, 91000 Evry, France.
Grégory CedroneGenethon, 91000 Evry, France.
Cladice Varela MoreiraGenethon, 91000 Evry, France.
Amandine LancelotGenethon, 91000 Evry, France.
Christophe GeorgerGenethon, 91000 Evry, France.
Aude LafouxNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France; Therassay Platform, Capacités, Nantes Université, 44200 Nantes, France.
Célia CouziniéNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France; Capacités Biotherapeutics Solutions (CBS), Capacités, Nantes Université, 44200 Nantes, France.
David AugereauNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France; Centre de Boisbonne, Oniris, INSERM, 44300 Nantes, France.
Corinne JounierNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France; Centre de Boisbonne, Oniris, INSERM, 44300 Nantes, France.
Agnès HivonnaitNantes Université, CNRS, INSERM, l'Institut du Thorax, 44007 Nantes, France.
Estelle CreoffGenethon, 91000 Evry, France.
Stéphanie BlaieGenethon, 91000 Evry, France.
Corinne HuchetNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France; Therassay Platform, Capacités, Nantes Université, 44200 Nantes, France.
Oumeya AdjaliNantes Université, CHU de Nantes, INSERM UMR 1089, Translational Gene Therapy Laboratory (TaRGeT), 44200 Nantes, France.
Nathalie DanieleGenethon, 91000 Evry, France.
Gérald PerretGenethon, 91000 Evry, France.
Serge BraunGenethon, 91000 Evry, France; AFM Telethon, 91000 Evry, France. Electronic address: sbraun@afm-telethon.fr.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recombinant adeno-associated virus (rAAV) vectors encoding microdystrophin (MD) are a promising treatment for Duchenne muscular dystrophy (DMD). GNT0004, an rAAV2/8 vector expressing MD1, is currently being tested in patients with DMD. Here, we explored supra-optimal intravenous doses of GNT0004 (2.1 × 10

Indexed as

Arrhythmias, CardiacDeath, Sudden, CardiacDystrophinGenetic TherapyMuscular Dystrophy, DuchenneAnimalsDependovirusDisease Models, AnimalGene ExpressionGenetic VectorsGenotypeHumansMaleMuscle, SkeletalMyocardiumRatsDystrophincardiac arrhythmiaDMD(mdx) ratDuchenne muscular dystrophygene therapymicrodystrophinoverexpressionrAAVsudden death

Identifiers

PMID41137396
PMCPMC12925808

What Socratic holds

Textmetadata
LicenceCC BY
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.