Abstract
Expanding knowledge about the crucial roles of microRNAs (miRNAs) in human diseases has led to the idea that miRNAs may be novel, promising therapeutic targets against various pathological conditions. The recent success of a human clinical trial using anti-miR-122 oligonucleotides against chronic hepatitis C virus has paved the way for this approach. In this review, we summarize briefly the current status of clinical trials of miRNA-targeting therapy and several representative preclinical trials against hepato-gastrointestinal carcinoma. In addition, we describe the currently available technologies for modification and delivery of oligonucleotides, which are essential in providing efficient, specific and safe approaches to targeting miRNAs.
Highlights
The expression and functional importance of non-coding RNAs, such as long ncRNAs and microRNAs, in various human diseases has been reported extensively [1,2,3]
In a xenograft model, overexpressed miR-7 effectively repressed tumor growth and decreased metastasis to the lung. These findings indicate that miR-7 functions as a tumor suppressor and plays a substantial role in inhibiting the tumorigenesis and reversing the metastasis of hepatocellular carcinoma (HCC) through the PI3K/Akt/mammalian target of rapamycin (mTOR)-signaling pathway
Along with recent discoveries of the diverse effects of miRNAs in biological systems, miRNA-mediated intervention is a promising avenue for the development of novel therapeutics against human diseases
Summary
The expression and functional importance of non-coding RNAs (ncRNAs), such as long ncRNAs and microRNAs, in various human diseases has been reported extensively [1,2,3]. These reports indicate that LNA-based anti-miRs can achieve efficient silencing of endogenous miRNA function in mammals and primates, which supports the application of anti-miRNA therapy to other human diseases These preclinical results led to the development of miravirsen, a LNA-modified DNA phosphorothioate antisense oligonucleotide against miR-122, as the first miRNAtargeting drug for clinical use [29]. In a xenograft model, overexpressed miR-7 effectively repressed tumor growth and decreased metastasis to the lung These findings indicate that miR-7 functions as a tumor suppressor and plays a substantial role in inhibiting the tumorigenesis and reversing the metastasis of HCC through the PI3K/Akt/mTOR-signaling pathway. Given these results, miR-7 may be a potential therapeutic or diagnostic/ prognostic target for treating HCC [49]. Introduction of miR-520e suppresses the growth of HCC cells in vitro by targeting NF-κB-inducing kinase (NIK), which is involved
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