title: Adeno-associated virus-mediated microRNA delivery and therapeutics.
abstract: MicroRNAs (miRNAs) are 20 to 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 the authors review currently used methods of altering miRNA activity in vivo. They 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.
authors: Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts.
authors: Jun
authors: J
authors: Xie
authors: Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts.
authors: Daniel Robert
authors: DR
authors: Burt
authors: Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts.
authors: Guangping
authors: G
authors: Gao
mesh_terms: D000229:Dependovirus; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D035683:MicroRNAs / Q000037:antagonists & inhibitors / Q000627:therapeutic use*
chemical_list: D035683:MicroRNAs
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