Related Experiment Video

Updated: Oct 31, 2025

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells
07:23

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

Published on: May 30, 2025

798

Towards a druggable epitranscriptome: Compounds that target RNA modifications in cancer

María Berdasco1,2, Manel Esteller3,4,5,6

  • 1Epigenetic Therapies Group, Experimental and Clinical Hematology Program (PHEC), Josep Carreras Leukaemia Research Institute, Barcelona, Spain.

British Journal of Pharmacology
|June 29, 2021
PubMed

Insights

Epitranscriptomics studies RNA modifications, revealing their role in cancer. This review explores targeting these RNA marks with drugs for cancer treatment, highlighting current challenges and future potential.

Area of Science:

  • Biochemistry and Molecular Biology
  • Cancer Research
  • Pharmacology

Background:

  • Epitranscriptomics investigates biochemical modifications of RNA, providing insights into cellular processes.
  • Recent advancements enable characterization and quantification of RNA modifications.
  • Aberrant epitranscriptomes are increasingly linked to human diseases, particularly cancer.

Purpose of the Study:

  • To review key RNA modifications, focusing on mRNA.
  • To summarize evidence of RNA modification dysregulation in cancer.
  • To provide an overview of drug discovery efforts targeting the epitranscriptome.

Main Methods:

  • Literature review of epitranscriptomic research.
  • Analysis of scientific evidence linking RNA modifications to cancer.
  • Survey of current drug discovery strategies for epitranscriptomic targets.

Main Results:

  • Detailed description of principal RNA modifications, especially on mRNA.
  • Compilation of data showing RNA modification alterations in various cancers.
  • Overview of the current landscape of small molecule compounds targeting the epitranscriptome.

Conclusions:

  • Epitranscriptomics presents potential therapeutic targets for cancer.
  • Targeting RNA modifications with low molecular weight compounds is a promising, yet challenging, area.
  • Further advancements in chemical biology and drug development are crucial for clinical translation.
Keywords:
A-to-I-editingRNA methylationcancerepitranscriptomicspseudouridinesmall-molecule inhibitorstherapy

More Related Videos

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes09:44

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes

Published on: March 3, 2015

9.7K
Using RNA-sequencing to Detect Novel Splice Variants Related to Drug Resistance in In Vitro Cancer Models09:58

Using RNA-sequencing to Detect Novel Splice Variants Related to Drug Resistance in In Vitro Cancer Models

Published on: December 9, 2016

14.0K

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...
3.3K
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...
22.6K
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
8.0K
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...
26.9K
RNA Editing02:23

RNA Editing

RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
9.3K
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...
6.6K