Evidence map›Paper›PMID 39967852›Full record

ArticleMolecular therapy. Nucleic acids2025

Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells.

Saeed Anwar, Rohini Roy Roshmi, Stanley Woo, Umme Sabrina Haque, Joshua James Arthur Lee, William John Duddy, Anne Bigot, Rika Maruyama, Toshifumi Yokota

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. 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

9 authors.

Saeed AnwarDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
Rohini Roy RoshmiDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
Stanley WooDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
Umme Sabrina HaqueNeuroscience and Mental Health Institute, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2R3, Canada.
Joshua James Arthur LeeDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
William John DuddyPersonalised Medicine Centre, School of Medicine, Ulster University, BT47 6SB Derry-Londonderry, UK.
Anne BigotCentre de Recherche en Myologie, Institut de Myologie, Sorbonne Université-L'Institut National de la Santé et de la Recherche Médicale (INSERM), 75651 Paris Cedex, France.
Rika MaruyamaDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
Toshifumi YokotaDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Dysferlinopathies are debilitating autosomal recessive muscular dystrophies caused by mutations in the

Indexed as

antisense oligonucleotideASOdysferlindysferlinopathyexon skippingLGMDR2limb-girdle muscular dystrophy recessive type 2membrane resealingMT: Oligonucleotides: Therapies and Applicationsphosphorodiamidate morpholino oligomerPMO

Identifiers

PMID39967852
PMCPMC11834094

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.