AAV-mediated miRNA Delivery and Therapeutics #MMPMID25632938
Xie J; Burt DR; Gao G
Semin Liver Dis 2015[Feb]; 35 (1): 81-8 PMID25632938show ga
MicroRNAs are 20-24 nt long, single-stranded RNAs that repress gene expression. Dysregulation of miRNA expression is associated with many human diseases. Modulating the level of endogenous miRNA alters gene profiling and can achieve therapeutic benefits. Here, we reviewed currently used methods of altering miRNA activity in vivo. We focus on the delivery of miRNAs and miRNA inhibitors using recombinant adeno-associated virus (rAAV). In general, rAAV-mediated miRNA inhibition or overexpression provides a simple, efficient and informative way to study miRNA function in mammals. This method also provides the opportunity to explore potential miRNA therapeutics for many diseases.