Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene Therapy
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.
Key wordsmiRNA Muscle Cardiac Gene expression Posttranscriptional regulation Gene therapy
We thank members of the Wang laboratory for discussion and support. Research in the Wang lab was supported by the March of Dimes Birth Defect Foundation, National Institutes of Health and Muscular Dystrophy Association. DZ Wang is an established investigator of the American Heart Association.
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