Non-Coding RNAs in Muscle Dystrophies

International Journal of Molecular Sciences

Daniela Erriquez¹  Giovanni Perini¹ 

[1] Department of Pharmacy and Biotechnology, University of Bologna, Bologna 40126, Italy; E-Mail:

关键词: microRNAs (miRNAs);    long non-coding RNAs (lncRNAs);    Duchenne muscular dystrophy (DMD);    Becker muscular dystrophy (BMD);    Myotonic dystrophies (DM1 and DM2);    Facioscapulohumeral dystrophy (FSHD);   

DOI  :  10.3390/ijms141019681

来源: mdpi

【 摘 要 】

ncRNAs are the most recently identified class of regulatory RNAs with vital functions in gene expression regulation and cell development. Among the variety of roles they play, their involvement in human diseases has opened new avenues of research towards the discovery and development of novel therapeutic approaches. Important data come from the field of hereditary muscle dystrophies, like Duchenne muscle dystrophy and Myotonic dystrophies, rare diseases affecting 1 in 7000–15,000 newborns and is characterized by severe to mild muscle weakness associated with cardiac involvement. Novel therapeutic approaches are now ongoing for these diseases, also based on splicing modulation. In this review we provide an overview about ncRNAs and their behavior in muscular dystrophy and explore their links with diagnosis, prognosis and treatments, highlighting the role of regulatory RNAs in these pathologies.

【 授权许可】

CC BY   
© 2013 by the authors; licensee MDPI, Basel, Switzerland