Noncoding RNAs and Duchenne Muscular Dystrophy

Abstract

Noncoding RNAs (ncRNAs) such as miRNAs and long noncoding RNAs modulate gene transcription in response to environmental stressors and other stimuli. A role for ncRNAs in muscle pathologies has been demonstrated and further evidence suggests that ncRNAs also play a role in Duchenne muscular dystrophy (DMD). Studies investigating the differential expression of miRNAs in biological fluids between DMD patients and models of dystrophin deficiency (the MDX mouse model, canine models of DMD) and controls have been published, as these have a role in fibrosis. Long noncoding RNAs are differentially expressed in DMD patients and may, in part, have a mechanism of action via targeting of miRNAs. Although many of these recent findings need to be confirmed, ncRNAs may prove to be useful as potential biomarkers of disease. However, their use as therapeutic targets in DMD remains unclear.

Keywords::

• Duchenne muscular dystrophy
• GRMD dog
• lncRNA
• MDX mice
• miRNA

Financial & competing interests disclosure

MM Perry is funded by the Association Francais contre les Myopathies, F Muntoni is supported by the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. MM Perry is a member of Interuniversity Attraction Poles Program-Belgian State-Belgian Science Policy - project P7/30. The support of the Muscular Dystrophy UK to the Dubowitz Neuromuscular Centre is also gratefully acknowledged. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Additional information

Funding

MM Perry is funded by the Association Francais contre les Myopathies, F Muntoni is supported by the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. MM Perry is a member of Interuniversity Attraction Poles Program-Belgian State-Belgian Science Policy - project P7/30. The support of the Muscular Dystrophy UK to the Dubowitz Neuromuscular Centre is also gratefully acknowledged. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. No writing assistance was utilized in the production of this manuscript.