Evidence map›Paper›PMID 21251213›Full record

ArticleJournal of cellular and molecular medicine2011

Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish.

Joachim Berger, Silke Berger, Arie S Jacoby, Steve D Wilton, Peter D Currie

Open access · bronzeAbstract readEvaluation Study
In one paragraph

Article in Journal of cellular and molecular medicine, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
1.6field-weighted citation impact, top 17% of its field
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

25 citing papers in PubMed, 45 citations in OpenAlex.

  1. Article
  2. Review
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  4. Article
  5. Loss of Sec-1 Family Domain-Containing 1 (Journal of cardiovascular development and disease · 2023
    Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Effect of Ataluren on dystrophin mutations.Journal of cellular and molecular medicine · 2020
    Article
  11. Review
  12. Zebrafish models of sarcopenia.Disease models & mechanisms · 2020
    Review
  13. Review
  14. Article
  15. Article
  16. Review
  17. Article
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  19. Article
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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 at 3 institutions in 1 country.

Joachim BergerAustralian Regenerative Medicine Institute, Monash University, Clayton, Victoria, Australia.
Silke Berger
Arie S Jacoby
Steve D Wilton
Peter D Currie
Australian Regenerative Medicine Institute · AUThe University of Western Australia · AUVictor Chang Cardiac Research Institute · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne muscular dystophy (DMD) is a severe muscle wasting disease caused by mutations in the dystrophin gene. By utilizing antisense oligonucleotides, splicing of the dystrophin transcript can be altered so that exons harbouring a mutation are excluded from the mature mRNA. Although this approach has been shown to be effective to restore partially functional dystrophin protein, the level of dystrophin protein that is necessary to rescue a severe muscle pathology has not been addressed. As zebrafish dystrophin mutants (dmd) resemble the severe muscle pathology of human patients, we have utilized this model to evaluate exon skipping. Novel dmd mutations were identified to enable the design of phenotype rescue studies via morpholino administration. Correlation of induced exon-skipping efficiency and the level of phenotype rescue suggest that relatively robust levels of exon skipping are required to achieve significant therapeutic ameliorations and that pre-screening analysis of exon-skipping drugs in zebrafish may help to more accurately predict clinical trials for therapies of DMD.

Indexed as

AnimalsAnimals, Genetically ModifiedDystrophinExonsHumansMuscular Dystrophy, DuchennePhenotypeReal-Time Polymerase Chain ReactionRNA, MessengerZebrafishDystrophinRNA, Messenger

Identifiers

PMID21251213
PMCPMC4373433
OpenAlexW2135265213

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.