Elmén Joacim, Lindow Morten, Silahtaroglu Asli, Bak Mads, Christensen Mette, Lind-Thomsen Allan, Hedtjärn Maj, Hansen Jens Bo, Hansen Henrik Frydenlund, Straarup Ellen Marie, McCullagh Keith, Kearney Phil, Kauppinen Sakari
Santaris Pharma, Bøge Allé 3, DK-2970 Hørsholm, Denmark.
Nucleic Acids Res. 2008 Mar;36(4):1153-62. doi: 10.1093/nar/gkm1113. Epub 2007 Dec 23.
MicroRNA-122 (miR-122) is an abundant liver-specific miRNA, implicated in fatty acid and cholesterol metabolism as well as hepatitis C viral replication. Here, we report that a systemically administered 16-nt, unconjugated LNA (locked nucleic acid)-antimiR oligonucleotide complementary to the 5' end of miR-122 leads to specific, dose-dependent silencing of miR-122 and shows no hepatotoxicity in mice. Antagonism of miR-122 is due to formation of stable heteroduplexes between the LNA-antimiR and miR-122 as detected by northern analysis. Fluorescence in situ hybridization demonstrated uptake of the LNA-antimiR in mouse liver cells, which was accompanied by markedly reduced hybridization signals for mature miR-122 in treated mice. Functional antagonism of miR-122 was inferred from a low cholesterol phenotype and de-repression within 24 h of 199 liver mRNAs showing significant enrichment for miR-122 seed matches in their 3' UTRs. Expression profiling extended to 3 weeks after the last LNA-antimiR dose revealed that most of the changes in liver gene expression were normalized to saline control levels coinciding with normalized miR-122 and plasma cholesterol levels. Combined, these data suggest that miRNA antagonists comprised of LNA are valuable tools for identifying miRNA targets in vivo and for studying the biological role of miRNAs and miRNA-associated gene-regulatory networks in a physiological context.
微小RNA-122(miR-122)是一种在肝脏中大量表达的特异性微小RNA,与脂肪酸和胆固醇代谢以及丙型肝炎病毒复制有关。在此,我们报告称,一种经全身给药的、与miR-122 5'端互补的16核苷酸、未缀合的锁核酸(LNA)抗微小RNA寡核苷酸可导致miR-122发生特异性、剂量依赖性沉默,且在小鼠中未显示出肝毒性。通过Northern分析检测发现,miR-122的拮抗作用是由于LNA-抗微小RNA与miR-122之间形成了稳定的异源双链体。荧光原位杂交显示LNA-抗微小RNA被小鼠肝细胞摄取,同时在接受治疗的小鼠中成熟miR-122的杂交信号明显减少。从低胆固醇表型以及199个肝脏mRNA在24小时内的去抑制作用推断出miR-122的功能拮抗作用,这些mRNA在其3'非翻译区(UTR)中显示出与miR-122种子匹配的显著富集。在最后一次给予LNA-抗微小RNA剂量后3周进行的表达谱分析表明,肝脏基因表达的大多数变化恢复到了生理盐水对照水平,这与miR-122和血浆胆固醇水平的正常化相一致。综合这些数据表明,由LNA组成的微小RNA拮抗剂是在体内鉴定微小RNA靶点以及在生理背景下研究微小RNA和微小RNA相关基因调控网络生物学作用的有价值工具。