Noncoding RNAs and Duchenne muscular dystrophy.
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.
The Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guildford Street, London, WC1N 1EH, UK.
Mark M
MM
Perry
The Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guildford Street, London, WC1N 1EH, UK.
Francesco
F
Muntoni
D000818:Animals; D004195:Disease Models, Animal; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics*; D022661:RNA, Untranslated
D022661:RNA, Untranslated
Duchenne muscular dystrophy; GRMD dog; MDX mice; lncRNA; miRNA