Related Experiment Video

Updated: Apr 27, 2026

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
09:06

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method

Published on: October 7, 2025

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Therapeutic targeting of microRNAs: current status and future challenges

Zhonghan Li1, Tariq M Rana2

  • 1Drug Safety, Research &Development, Pfizer, 1 Burtt Road, Andover, Massachusetts 01845, USA.

Nature Reviews. Drug Discovery
|July 12, 2014
PubMed

Insights

Chemically modified antisense oligonucleotides can inhibit microRNAs (miRNAs), offering potential new therapies for diseases like cancer. This review covers their design, delivery, and clinical progress.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biochemistry

Background:

  • MicroRNAs (miRNAs) are small non-coding RNAs regulating gene expression.
  • Dysregulation of miRNAs is implicated in human diseases, including cancer and autoimmune disorders.
  • Antisense oligonucleotides offer a method to inhibit miRNA function.

Purpose of the Study:

  • To review the design and performance of chemically modified miRNA-targeting antisense oligonucleotides.
  • To discuss in vivo delivery strategies for these therapeutic oligonucleotides.
  • To analyze challenges and clinical progress in miRNA-targeting therapeutics.

Main Methods:

  • Review of current literature on miRNA-targeting antisense oligonucleotides.
  • Analysis of chemical modifications, delivery systems, and clinical trial data.
  • Evaluation of specificity and efficacy of therapeutic oligonucleotides in vivo.

Main Results:

  • Chemically modified antisense oligonucleotides demonstrate efficient and specific inhibition of miRNA function.
  • Various in vivo delivery strategies are being explored to enhance therapeutic efficacy.
  • Ongoing challenges include ensuring specificity and efficacy in vivo.

Conclusions:

  • Antisense oligonucleotides targeting miRNAs hold significant therapeutic potential for various diseases.
  • Further research and development are needed to overcome challenges in delivery and specificity.
  • Clinical development of miRNA-targeting therapeutics is progressing, with promising early results.

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