Evidence map›Paper›PMID 40720023›Full record

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

In Vivo Evaluation of Single- and Multi-Exon-Skipping in mdx52 Mice.

Chaitra Sathyaprakash, Eri Takeuchi, Hotake Takizawa, Yuki Ashida, Tsukasa Tominari, Yusuke Echigoya, Toshifumi Yokota, Yoshitsugu Aoki

Abstract read
PubMed Publisher
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. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

8 authors.

Chaitra SathyaprakashDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Eri TakeuchiDepartment 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.
Yuki AshidaDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Tsukasa TominariDepartment 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, Faculty of Medicine and Dentistry, University of Alberta, 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

Exon-skipping therapy is an emerging approach that uses synthetic DNA-like molecules called antisense oligonucleotides (ASOs) to splice out frame-disrupting regions of mRNA, restoring the reading frame, and produce truncated yet functional proteins. Phosphorodiamidate morpholino oligomers (PMOs) are one of the safest among therapeutic ASOs for patients and have recently been approved under the accelerated approval program by the US Food and Drug Administration (FDA) as the first ASO-based drug for Duchenne muscular dystrophy (DMD). Multi-exon-skipping utilizing ASOs can theoretically treat 80-90% of patients with DMD. Here, we describe the systemic delivery of a cocktail of ASOs to skip exon 51 and exons 45 to 55 in the mdx52 mouse, an exon 52 deletion model of DMD produced by gene targeting, and the evaluation of their efficacies in vivo.

Indexed as

DystrophinExonsGenetic TherapyMuscular Dystrophy, DuchenneOligonucleotides, AntisenseAnimalsDisease Models, AnimalMiceMice, Inbred mdxMorpholinosDystrophinMorpholinosOligonucleotides, AntisenseBecker muscular dystrophy (BMD)Duchenne muscular dystrophy (DMD)DystrophinEteplirsen (Exondys 51)Exon-skippingmdxmdx52Multi-exon-skippingPhosphorodiamidate morpholino oligomer (PMO)Vivo-morpholinos (vPMOs)

Identifiers

PMID40720023

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.