Correction of genetic disease by making sense from nonsense.

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.

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.

Who cites it

20 citing papers in PubMed.

1. Balancing Nonsense Mutation Readthrough and Toxicity of Designer Aminoglycosides for Treatment of Genetic Diseases.ACS medicinal chemistry letters · 2023
   Article
2. Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy.Frontiers in medicine · 2022
   Review
3. "Betwixt Mine Eye and Heart a League Is Took": The Progress of Induced Pluripotent Stem-Cell-Based Models of Dystrophin-Associated Cardiomyopathy.International journal of molecular sciences · 2020
   Review
4. Dystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision Medicine.Journal of clinical medicine · 2018
   Review
5. UPF1 silenced cellular model systems for screening of read-through agents active on βBMC biotechnology · 2018
   Article
6. Structure-Activity Relationship Studies of 3-epi-Deoxynegamycin Derivatives as Potent Readthrough Drug Candidates.ACS medicinal chemistry letters · 2015
   Article
7. High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics.Biology · 2014
   Review
8. When Proteins Start to Make Sense: Fine-tuning Aminoglycosides for PTC Suppression Therapy.MedChemComm · 2014
   Article
9. Translational read-through of a nonsense mutation causing Bartter syndrome.Journal of Korean medical science · 2013
   Article
10. Cisplatin and aminoglycoside antibiotics: hearing loss and its prevention.Anatomical record (Hoboken, N.J. : 2007) · 2012
    Review
11. The GABRG2 nonsense mutation, Q40X, associated with Dravet syndrome activated NMD and generated a truncated subunit that was partially rescued by aminoglycoside-induced stop codon read-through.Neurobiology of disease · 2012
    Article
12. USH1G with unique retinal findings caused by a novel truncating mutation identified by genome-wide linkage analysis.Molecular vision · 2012
    Article
13. New developments in aminoglycoside therapy and ototoxicity.Hearing research · 2011
    Review
14. Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Molecular therapy : the journal of the American Society of Gene Therapy · 2011
    Review
15. Ex vivo treatment with a novel synthetic aminoglycoside NB54 in primary fibroblasts from Rett syndrome patients suppresses MECP2 nonsense mutations.PloS one · 2011
    Article
16. Translational errors: from yeast to new therapeutic targets.FEMS yeast research · 2010
    Review
17. Molecular therapeutic strategies targeting Duchenne muscular dystrophy.Journal of child neurology · 2010
    Review
18. In vitro readthrough of termination codons by gentamycin in the Stüve-Wiedemann Syndrome.European journal of human genetics : EJHG · 2010
    Article
19. Development of K562 cell clones expressing beta-globin mRNA carrying the beta039 thalassaemia mutation for the screening of correctors of stop-codon mutations.Biotechnology and applied biochemistry · 2009
    Article
20. Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations.Journal of medicinal chemistry · 2009
    Article

Corrections and comments

• Comment on
  Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice.1999

Authors and funding

1 author.

Funding

No grant is acknowledged in the PubMed record.

Abstract

PubMed holds no abstract for this paper.

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