First Author | Su J | Year | 2011 |
Journal | Nucleic Acids Res | Volume | 39 |
Issue | 6 | Pages | e38 |
PubMed ID | 21212128 | Mgi Jnum | J:173723 |
Mgi Id | MGI:5050052 | Doi | 10.1093/nar/gkq1307 |
Citation | Su J, et al. (2011) Silencing microRNA by interfering nanoparticles in mice. Nucleic Acids Res 39(6):e38 |
abstractText | MicroRNAs (miRNAs) are small endogenous non-coding RNAs that regulate post-transcriptional gene expression and are important in many biological processes. Disease-associated miRNAs have been shown to become potential targets for therapeutic intervention. Functions of miRNAs can be inhibited by using antisense oligonucleotides, called anti-miRs, complimentary to the miRNA sequences. Here, we show that systemic delivery of a chemically stabilized anti-miR-122 complexed with interfering nanoparticles (iNOPs) effectively silences the liver-expressed miR-122 in mice. Intravenous administration of 2 mg kg(-1) chemically modified anti-miR-122 complexed with iNOP-7 resulted in 83.2 +/- 3.2% specific silencing of miR-122, which was accompanied by regulating gene expression in liver and lowering of plasma cholesterol. The specific silencing of miR-122 was long lasting and did not induce an immune response. Our results demonstrate that iNOPs can successfully deliver anti-miR to specifically target and silence miRNA in clinically acceptable and therapeutically affordable doses. |