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RNA-based medicine: from molecular mechanisms to therapy.

Anke SparmannJörg Vogel
Published in: The EMBO journal (2023)
RNA-based therapeutics have the potential to revolutionize the treatment and prevention of human diseases. While early research faced setbacks, it established the basis for breakthroughs in RNA-based drug design that culminated in the extraordinarily fast development of mRNA vaccines to combat the COVID-19 pandemic. We have now reached a pivotal moment where RNA medicines are poised to make a broad impact in the clinic. In this review, we present an overview of different RNA-based strategies to generate novel therapeutics, including antisense and RNAi-based mechanisms, mRNA-based approaches, and CRISPR-Cas-mediated genome editing. Using three rare genetic diseases as examples, we highlight the opportunities, but also the challenges to wide-ranging applications of this class of drugs.
Keyphrases
  • crispr cas
  • genome editing
  • nucleic acid
  • small molecule
  • endothelial cells
  • primary care
  • gene expression
  • emergency department
  • stem cells
  • dna methylation
  • copy number
  • bone marrow
  • climate change
  • drug induced
  • adverse drug