Coenen-Stass, AML;
Wood, MJA;
Roberts, TC;
(2017)
Biomarker Potential of Extracellular miRNAs in Duchenne Muscular Dystrophy.
Trends in Molecular Medicine
, 23
(11)
pp. 989-1001.
10.1016/j.molmed.2017.09.002.
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Abstract
miRNAs are small, noncoding RNAs that not only regulate gene expression within cells, but might also constitute promising extracellular biomarkers for a variety of pathologies, including the progressive muscle-wasting disorder Duchenne Muscular Dystrophy (DMD). A set of muscle-enriched miRNAs, the myomiRs (miR-1, miR-133, and miR-206) are highly elevated in the serum of patients with DMD and in dystrophin-deficient animal models. Furthermore, circulating myomiRs might be used as pharmacodynamic biomarkers, given that their levels can be restored towards wild-type levels following exon skipping therapy in dystrophic mice. The relationship between muscle pathology and extracellular myomiR release is complex, and incompletely understood. Here, we discuss current progress leading towards the clinical utility of extracellular miRNAs as putative DMD biomarkers, and their possible contribution to muscle physiology.
| Type: | Article |
|---|---|
| Title: | Biomarker Potential of Extracellular miRNAs in Duchenne Muscular Dystrophy |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.molmed.2017.09.002 |
| Publisher version: | http://doi.org/10.1016/j.molmed.2017.09.002 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Biomarkers, microRNA, extracellular vesicles, extracellular RNA, exosomes, Duchenne Muscular Dystrophy |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10042776 |
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