RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models.

Alex Chassin, Hiroya Ono, Yuki Ashida, Michihiro Imamura, Yoshitsugu Aoki

Abstract readReview

In one paragraph

Review in International journal of molecular sciences, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it

0cells of the map it votes in

3citing 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

3 citing papers in PubMed.

Beyond Coding Variants: RNA-Level Mechanisms in Human Disease and Precision Therapeutics.Genes · 2026
Review
RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.Biomolecules · 2026
Review
From Variant Interpretation to Biomarker Translation: Multi-omics Integration in Inherited Neuromuscular Diseases.Human mutation · 2026
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

5 authors.

Alex ChassinDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, Tokyo 187-8502, Japan.

Hiroya OnoDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, Tokyo 187-8502, Japan.ORCID 0000-0002-8439-5657

Yuki AshidaDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, Tokyo 187-8502, Japan.

Michihiro ImamuraDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, Tokyo 187-8502, Japan.

Yoshitsugu AokiDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, Tokyo 187-8502, Japan.ORCID 0000-0002-9038-7506

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disease (NMD) caused by loss-of-function mutations in the

Indexed as

ExonsGenetic TherapyMuscular Dystrophy, DuchenneRNA EditingAnimalsDisease Models, AnimalDystrophinHumansOligonucleotides, AntisenseDystrophinOligonucleotides, AntisenseADARanimal modelsantibody–oligonucleotide conjugateantisense oligonucleotidesDuchenne muscular dystrophyexon skippinggenome editingmicrominipigpeptide-conjugated PMOphosphorodiamidate morpholino oligomerRNA editingRNA therapeutics

Identifiers

PMID41898615

DOI10.3390/ijms27062755

PMCPMC13027266

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.