RNA modifications in cancer: from biological insights to therapeutic targets

Yutao Zhao1, Chuan He1

  • 1Department of Chemistry, Department of Biochemistry and Molecular Biology, and Institute for Biophysical Dynamics, The University of Chicago, Chicago, IL 60637, USA; Howard Hughes Medical Institute, The University of Chicago, Chicago, IL 60637, USA.

PubMed

Insights

RNA modifications like N6-methyladenosine (m6A) regulate gene expression and impact diseases. Targeting these epitranscriptomic marks shows therapeutic potential in preclinical cancer models.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biochemistry

Background:

  • RNA modifications are crucial for gene expression regulation and cellular homeostasis.
  • Key modifications like N6-methyladenosine (m6A) and pseudouridine (Ψ) affect transcription, splicing, stability, and translation.
  • Epitranscriptomics, the study of RNA modifications, has rapidly advanced due to high-throughput sequencing.

Purpose of the Study:

  • To provide a historical overview of epitranscriptomics.
  • To examine the biological functions of RNA modifications, particularly in cancer development.
  • To summarize recent therapeutic strategies targeting RNA modification pathways.

Main Methods:

  • Review of key discoveries and technological advancements in epitranscriptomics.
  • Analysis of literature on the roles of RNA modifications in cancer.
  • Summary of preclinical studies on small-molecule inhibitors targeting RNA modification effectors.

Main Results:

  • RNA modifications play significant roles in various cellular processes.
  • These modifications are implicated as drivers in cancer development.
  • Small-molecule inhibitors targeting RNA modification pathways show promise in preclinical cancer models.

Conclusions:

  • RNA modifications are essential regulators of gene expression with implications for human diseases.
  • Targeting the epitranscriptome represents a promising therapeutic avenue for cancer treatment.

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