miRNA-based Drugs

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miRNA-based Drugs

microRNAs (miRNAs) are short, non-coding, single-stranded RNA molecules that play a critical role in several biological processes such as immune modulation, metabolic control, neuronal development, cell cycle, muscle differentiation, and stem cell differentiation. Recent studies show that miRNAs targeting pathways of human disease provide a new and potential powerful candidate for therapeutic intervention against various pathological conditions. In addition, it's reported that miRNAs function as oncogenes (oncomiRs) or tumor suppressors and their expression is often dysregulated in tumors.

microRNA (miRNA)-based drugs can be divided into miRNA mimics and inhibitors of miRNAs (also known as antimiRs). At present, several miRNA-based drugs have reached clinical development, including a mimic of the tumor suppressor miRNA miR-34, which reached phase I clinical trials for treating cancer, and antimiRs targeted at miR-122, which reached phase II trials for treating hepatitis.

  • miRNA mimics

miRNA mimics are synthetic double-stranded small RNA molecules that match the corresponding miRNA sequence and therefore functionally aim to replenish the lost miRNA expression in diseases.

Antisense mechanisms of miRNA mimic Fig1. Antisense mechanisms of miRNA mimic.

  • miRNA antagonists

Also called miRNA inhibitors, miRNA antagonists are small, synthetically derived molecules, which have sequence complementary to target mature miRNAs. They are known to sequester target miRNAs and are used to suppress miRNA function.

Antisense mechanisms of miRNA antagonist Fig2. Antisense mechanisms of miRNA antagonist

The process of miRNA-based drugs discovery and development

miRNA-based Drugs

However, only a small number of miRNA-based drugs have moved in to clinical development since miRNAs were discovered. While substantial challenges remain in this area, including identification of the best miRNA targets for drug development, and the design of delivery vehicles that allow better drug stability and tissue-specific targeting. The genomics and proteomics tools now available to researchers, coupled with novel delivery technologies will enable miRNA-based drugs to become a viable option for the treatment of many diseases.

IntegrateRNA provides world-class microRNA services to accelerate your research. We have some of the best trained and most experienced subject matter experts in this field. We provide miRNA agomir/antagomir synthesis, miRNA sequencing, custom miRNA microarray and miRNA functional analysis services. If you have any questions, don't hesitate to contact us.

Reference:

  1. Rupaimoole R. et al. MicroRNA therapeutics: towards a new era for the management of cancer and other diseases. Nature Reviews Drug Discovery, 2017, 16(3):203-222.
For research use only. Not intended for any clinical use.
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