RNA-based therapeutics for neurological diseases

Research output: Contribution to JournalReview Articlepeer-review


RNA-based therapeutics have entered the mainstream with seemingly limitless possibilities to treat all categories of neurological disease. Here, common RNA-based drug modalities such as antisense oligonucleotides, small interfering RNAs, RNA aptamers, RNA-based vaccines and mRNA drugs are reviewed highlighting their current and potential applications. Rapid progress has been made across rare genetic diseases and neurodegenerative disorders, but safe and effective delivery to the brain remains a significant challenge for many applications. The advent of individualised RNA-based therapies for ultra-rare diseases is discussed against the backdrop of the emergence of this field into more common conditions such as Alzheimer’s disease and ischaemic stroke. There remains significant untapped potential in the use of RNA-based therapeutics for behavioural disorders and tumours of the central nervous system; coupled with the accelerated development expected over the next decade, the true potential of RNA-based therapeutics to transform the therapeutic landscape in neurology remains to be uncovered.
Original languageEnglish
Pages (from-to)176-190
Number of pages15
JournalRNA Biology
Issue number1
Early online date22 Jan 2022
Publication statusPublished - 22 Jan 2022


  • RNA
  • RNA therapeutics
  • Exon skipping
  • Antisense oligonucleotides
  • Neurological disease
  • siRNA
  • RNA aptamer
  • RNA vaccine
  • Molecular Biology
  • Cell Biology


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