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Antisense oligonucleotides and other genetic therapies made simple
  1. Alexander M Rossor1,2,
  2. Mary M Reilly1,
  3. James N Sleigh2
  1. 1 MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology and the National Hospital for Neurology and Neurosurgery, London, UK
  2. 2 Sobell Department of Motor Neuroscience and Movement Disorders, Institute of Neurology, University College London, London, UK
  1. Correspondence to Dr Alexander M Rossor; a.rossor{at}


Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors. This review provides a brief overview of the main types of genetic therapies, with a focus on antisense oligonucleotides (ASOs) and RNA interference (RNAi). We use examples of new genetic therapies for spinal muscular atrophy, Duchenne muscular dystrophy and familial amyloid polyneuropathy to highlight the different mechanisms of action of ASOs and RNAi.

  • neurogenetics
  • Rna interference
  • antisense oligonucleotides
  • viral gene therapy
  • genome editing

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  • Contributors AMR wrote the first draft of the manuscript. JNS designed the figures and revised the manuscript. MMR revised the manuscript.

  • Funding AMR is funded by a Wellcome Trust Postdoctoral Fellowship for Clinicians(110043/Z/15/Z). MMR is grateful to the Medical Research Council (MRC), MRC Centre grant (G0601943), and to the National Institutes of Neurological Diseases and Stroke and office of Rare Diseases (U54NS065712) for their support. The INC (U54NS065712) is a part of the NCATS Rare Diseases Clinical Research Network (RDCRN). RDCRN is an initiative of the Office of Rare Diseases Research (ORDR), NCATS, funded through a collaboration between NCATS and the NINDS. This research was also supported by the National Institute for Health Research University College London Hospitals Biomedical Research Centre. JNS is supported by a Wellcome Trust Sir Henry Wellcome Postdoctoral Fellowship [103191/A/13/Z]

  • Competing interests None declared.

  • Provenance and peer review Commissioned; externally peer reviewed. This paper was reviewed by Simon Hammans, Southampton, UK.

  • Correction notice This article has been corrected since it published Online First. The middle initial for author James Sleigh has been corrected to ‘N’.

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