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Semin Liver Dis 2015; 35(01): 081-088
DOI: 10.1055/s-0034-1397352
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Adeno-Associated Virus-Mediated MicroRNA Delivery and Therapeutics

Jun Xie
1   Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts
2   Microbiology and Physiology Systems, University of Massachusetts Medical School, Worcester, Massachusetts
,
Daniel Robert Burt
1   Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts
3   Saint Louis University School of Medical, St. Louis, Missouri
,
Guangping Gao
1   Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts
2   Microbiology and Physiology Systems, University of Massachusetts Medical School, Worcester, Massachusetts
4   State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, Sichuan, People's Republic of China
› Author Affiliations
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Publication History

Publication Date:
29 January 2015 (online)

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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.

Keywords

adeno-associated virus - in vivo gene delivery - microRNA inhibition - microRNA overexpression
 

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