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Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

Overview of attention for article published in Molecular Therapy - Nucleic Acids, December 2015
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About this Attention Score

  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (86th percentile)
  • Good Attention Score compared to outputs of the same age and source (66th percentile)

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3 X users
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3 patents

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Title
Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
Published in
Molecular Therapy - Nucleic Acids, December 2015
DOI 10.1038/mtna.2015.39
Pubmed ID
Authors

Silvana M G Jirka, Christa L Tanganyika-de Winter, Joke W Boertje-van der Meulen, Maaike van Putten, Monika Hiller, Rick Vermue, Peter C de Visser, Annemieke Aartsma-Rus

Abstract

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder typically caused by frame-shifting mutations in the DMD gene. Restoration of the reading frame would allow the production of a shorter but partly functional dystrophin protein as seen in Becker muscular dystrophy patients. This can be achieved with antisense oligonucleotides (AONs) that induce skipping of specific exons during pre-mRNA splicing. Different chemical modifications have been developed to improve AON properties. The 2'-deoxy-2'-fluoro (2F) RNA modification is attractive for exon skipping due to its ability to recruit ILF2/3 proteins to the 2F/pre-mRNA duplex, which resulted in enhanced exon skipping in spinal muscular atrophy models. In this study, we examined the effect of two different 2'-substituted AONs (2'-F phosphorothioate (2FPS) and 2'-O-Me phosphorothioate (2OMePS)) on exon skipping in DMD cell and animal models. In human cell cultures, 2FPS AONs showed higher exon skipping levels than their isosequential 2OMePS counterparts. Interestingly, in the mdx mouse model, 2FPS was less efficient than 2OMePS and suggested safety issues as evidenced by increased spleen size and weight loss. Our results do not support a clinical application for 2FPS AON.

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The data shown below were collected from the profiles of 3 X users who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 46 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 46 100%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 11 24%
Student > Master 7 15%
Researcher 7 15%
Student > Bachelor 6 13%
Other 4 9%
Other 5 11%
Unknown 6 13%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 13 28%
Agricultural and Biological Sciences 7 15%
Medicine and Dentistry 6 13%
Neuroscience 3 7%
Chemistry 2 4%
Other 5 11%
Unknown 10 22%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 11. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 27 September 2022.
All research outputs
#3,274,362
of 25,373,627 outputs
Outputs from Molecular Therapy - Nucleic Acids
#248
of 1,841 outputs
Outputs of similar age
#51,548
of 395,397 outputs
Outputs of similar age from Molecular Therapy - Nucleic Acids
#6
of 21 outputs
Altmetric has tracked 25,373,627 research outputs across all sources so far. Compared to these this one has done well and is in the 86th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 1,841 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 6.6. This one has done well, scoring higher than 85% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 395,397 tracked outputs that were published within six weeks on either side of this one in any source. This one has done well, scoring higher than 86% of its contemporaries.
We're also able to compare this research output to 21 others from the same source and published within six weeks on either side of this one. This one has gotten more attention than average, scoring higher than 66% of its contemporaries.