RNAi therapeutics and applications of microRNAs in cancer treatment

Keita Uchino1, Takahiro Ochiya, Fumitaka Takeshita

  • 1Division of Molecular and Cellular Medicine, National Cancer Center Research Institute, 1-1, Tsukiji 5-chome, Chuo-ku, Tokyo 104-0045, Japan.

Insights

RNA interference therapies show promise for cancer treatment. Researchers are developing advanced delivery systems for fragile RNA molecules and targeting microRNAs to advance nucleic acid drug applications.

Area of Science:

  • Biomedical Science
  • Molecular Biology
  • Oncology

Background:

  • RNA interference (RNAi) therapies are emerging as potent treatments for diseases, notably cancer.
  • Developing safe and efficient delivery systems for fragile small RNA molecules to target cells is a key challenge.
  • MicroRNAs (miRNAs), crucial regulators of cellular processes, are recognized as significant therapeutic targets in oncology.

Purpose of the Study:

  • To review RNAi-based therapeutics currently in clinical trials.
  • To discuss cutting-edge technologies for systemic delivery of nucleic acid drugs.
  • To explore the role of dysregulated microRNAs in human cancers and strategies for their therapeutic modulation.

Main Methods:

  • Overview of RNA interference-based therapeutics in clinical trials.
  • Discussion of systemic delivery technologies for nucleic acid drugs.
  • Focus on microRNA dysregulation in cancer and methods for controlling miRNA expression.

Main Results:

  • A dozen pre-clinical and clinical trials are investigating RNAi therapies using various delivery methods like nanoparticles and chemical modifications.
  • MicroRNAs have shown promise as therapeutic targets in pre-clinical cancer studies.
  • Diverse strategies are being developed to modulate endogenous microRNA levels.

Conclusions:

  • Advancements in RNA interference technologies and clinical trials are paving the way for practical applications of nucleic acid drugs.
  • Effective delivery systems are crucial for the success of RNAi-based therapies.
  • Targeting microRNAs offers a promising avenue for novel cancer treatments.

Related Concept Videos

MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...