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Nucleic Acid Therapeutics for "Undruggable" Cancer Targets: Mechanisms, Challenges, and Prospects - PubMed

2 hours ago
  • #Undruggable Targets
  • #Cancer Therapy
  • #Nucleic Acid Therapeutics
  • Nucleic acid therapeutics provide a new approach to target historically difficult-to-drug cancer proteins like Ras, MYC, and p53 by acting at the mRNA or genomic levels.
  • Various nucleic acid modalities, including ASOs, siRNAs, miRNAs, aptamers, and mRNA vaccines, are discussed for their mechanisms, such as transcript degradation and translational inhibition.
  • Key challenges for translation include delivery efficiency, safety profiling, and scalable manufacturing, with recent advances in nanocarrier platforms highlighted.
  • Emerging technologies and computational design are explored as future directions to enhance the development and application of these therapies.
  • This review underscores nucleic acid therapeutics as a paradigm shift enabling precise regulation of 'undruggable' cancer targets.