RNA-target Therapeutics



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RNA-target Therapeutics

Recent advances in biopharmaceuticals have expanded the range of therapeutic targets for a variety of human diseases. RNA-target therapies have received widespread attention for their potential to treat a variety of chronic and rare diseases, and address targets that have proven to be intractable to antibody and small-molecule approaches. Despite the many obstacles encountered in this process, more than 50 RNA or RNA-derived therapeutic agents have been clinically tested. These include oligonucleotides (ASO), small interfering RNAs (siRNAs), microRNAs, mRNAs and aptamers.

Figure 1. RNA-based strategies to generate new types of therapeutics in a variety of diseasesFigure 1. RNA-based strategies to generate new types of therapeutics in a variety of diseases

To date, RNA-targeting therapeutics are already being applied in various diseases including cancers, genetic disorders and viral infections.

  • Cancer

In terms of cancer targeting, therapeutic modalities have been developed involving either short non-coding RNAs (siRNA or miRNA) or IVT mRNA. RNA-based cancer treatment could be achieved by targeting diverse signal pathways related to tumor growth and spreading including carcinogenesis, cell cycle regulation, anti-apoptosis pathway, multidrug resistance, angiogenesis, and cancer metastasis.

  • Genetic disease

Many genetic diseases result from errors that lead to anomalies in transcription, splicing, or translation, for example, spinal muscular atrophy (SMA). Targeting the flawed protein products directly is often ineffective. In many cases, RNA-target therapeutics offer the prospect of resolving the inborn error by normalizing splicing or by suppressing or promoting translation.

  • Infectious disease

RNA-based therapeutics has been suggested as one of the most innovative approaches to derive an advance in the field of drug and vaccine development against the infectious disease. For example, siRNA-mediated therapeutics has been applied in targeting RNAs of virus, including respiratory syncytial virus (RSV), HIV-1, rotavirus, HBV, and influenza virus.

Challenges of RNA-target therapeutics:

  • Intracellular delivery across cell and endosomal membranes
  • Poor pharmacokinetic properties, partly due to urinary excretion and ubiquitous RNases
  • Activation of innate immune nucleic acid sensors
  • Off-target effects

The goal of the IntegrateRNA is to support basic and applied research in RNA biology to generate new and novel insights into the role of RNA in health and disease and provide new tools and targets for RNA research, diagnostics and therapies. For further information, please feel free to contact us.


  1. Hojeong, Shin. et al. Recent Advances in RNA Therapeutics and RNA Delivery Systems Based on Nanoparticles. Advanced Therapeutics, 2017.
  2. Lieberman, Judy. et al. Tapping the RNA world for therapeutics. Nature Structural & Molecular Biology, 2018.
  3. Wei, Yin, Mark, et al. Targeting RNA: A Transformative Therapeutic Strategy. Clinical and translational science, 2019.
For research use only. Not intended for any clinical use.
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