RNA therapeutics for neurological disease

Prog Mol Biol Transl Sci. 2024:203:165-180. doi: 10.1016/bs.pmbts.2024.01.005. Epub 2024 Jan 24.


Neurological disorders are the group of diseases that primarily affect the center nervous system, which could lead to a significant negative impact on the ability of learning new skills, speaking, breathing, walking, judging, making decision, and other essential living skills. In the last decade, neurological disorders have significantly increased their impact to our community and become the one of leading causes of disability and death. The World Health Organization has identified neurological disorders including Alzheimer's disease and other dementia as the health crisis for the modern life. Tremendous ongoing research efforts focus on understanding of disease genetics, molecular mechanisms and developing therapeutic interventions. Because of the urgent need of the effective therapeutics and the recent advances in the toolkits and understanding for developing more drug-like RNA molecules, there is a growing interest for developing RNA therapeutics for neurological disorders. This article will discuss genetics and mechanisms of neurological disorders and how RNA-based molecules have been used to develop therapeutics for this group of diseases, challenges of RNA therapeutics and future perspectives on this rising therapeutic intervention tool.

Keywords: AAV; CRISPR/Cas based therapeutics; RNA based molecules; antisense oligos; gene delivery; neurodegenerative disorders; neurological disorders; repeat expansion disorders.

MeSH terms

  • Alzheimer Disease*
  • Central Nervous System
  • Humans
  • Nervous System Diseases* / genetics
  • Nervous System Diseases* / therapy
  • RNA / genetics


  • RNA