Evidence map›Paper›PMID 40720040›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2025

Exon-Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.

Eri Takeuchi, Chaitra Sathyaprakash, Hotake Takizawa, Norio Motohashi, Yusuke Echigoya, Toshifumi Yokota, Yoshitsugu Aoki

Abstract read
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In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2025. 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. 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

7 authors.

Eri TakeuchiDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Chaitra SathyaprakashDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Hotake TakizawaDepartment of Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Norio MotohashiDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Yusuke EchigoyaLaboratory of Biomedical Science, Department of Veterinary Medicine, College of Bioresource Sciences, Nihon University, Fujisawa, Kanagawa, Japan.
Toshifumi YokotaDepartment of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, Edmonton, AB, Canada.
Yoshitsugu AokiDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan. tsugu56@ncnp.go.jp.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Phosphorodiamidate morpholino oligomer (PMO)-mediated exon-skipping is among the more promising approaches available for the treatment of several neuromuscular disorders, including Duchenne muscular dystrophy. The main weakness of this treatment arises from the low efficiency and sporadic nature of the delivery of neutrally charged PMOs into muscle fibres, the mechanism of which is unknown. Recently, using wild-type and dystrophic mdx52 mice, we showed that muscle fibres took up PMOs more efficiently during myotube formation. Interestingly, we detected PMO mainly in embryonic myosin heavy chain-positive regenerating fibres through in situ hybridisation. Next, we tested the therapeutic potential of PMOs in laminin-alpha2 (laminin-α2) chain-null dy

Indexed as

ExonsLamininMuscular DystrophiesOligonucleotides, AntisenseAnimalsDisease Models, AnimalGenetic TherapyMiceMice, Inbred mdxMorpholinosMuscle Fibers, SkeletalLamininlaminin alpha 2MorpholinosOligonucleotides, Antisensedy3K/dy3K mouseDystrophinEteplirsenExon-skippingLaminin-α2 chainMerosin-deficient congenital muscular dystrophy type 1A (MDC1A or LAMA2-Related muscular dystrophiesMuscular dystrophies: LAMA2-MD)NS-065/NCNP-01Phosphorodiamidate morpholino oligomer (PMO)Viltolarsen

Identifiers

PMID40720040

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.