title: Application of microRNA in cardiac and skeletal muscle disease gene therapy.
abstract: MicroRNAs (miRNAs) are a class of small ∼22 nt noncoding RNAs. miRNAs regulate gene expression at the posttranscriptional levels by destabilization and degradation of the target mRNA or by translational repression. Numerous studies have demonstrated that miRNAs are essential for normal mammalian development and organ function. Deleterious changes in miRNA expression play an important role in human diseases. We and others have previously reported several muscle-specific miRNAs, including miR-1/206, miR-133, and miR-208. These muscle-specific miRNAs are essential for normal myoblast differentiation and proliferation, and they have also been implicated in various cardiac and skeletal muscular diseases. miRNA-based gene therapies hold great potential for the treatment of cardiac and skeletal muscle disease(s). Herein, we introduce the methods commonly applied to study the biological role of miRNAs, as well as the techniques utilized to manipulate miRNA expression.
authors.affiliation: Cardiovascular Research Division, Department of Cardiology, Children's Hospital Boston, Harvard Medical School, 320 Longwood Avenue, Boston, MA, USA.
authors.forename: Zhan-Peng
authors.initials: ZP
authors.lastname: Huang
authors.forename: Ronald L
authors.initials: RL
authors.lastname: Neppl
authors.forename: Da-Zhi
authors.initials: DZ
authors.lastname: Wang
mesh_terms: D015152:Blotting, Northern; D016189:Dystrophin / Q000235:genetics; D005786:Gene Expression Regulation; D015316:Genetic Therapy; D006331:Heart Diseases / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D006801:Humans; D035683:MicroRNAs / Q000235:genetics* / Q000378:metabolism; D018482:Muscle, Skeletal / Q000473:pathology; D009135:Muscular Diseases / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D009206:Myocardium / Q000473:pathology; D012323:RNA Processing, Post-Transcriptional; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism
chemical_list: D016189:Dystrophin; D035683:MicroRNAs; D012333:RNA, Messenger