Antisense oligonucleotides: the next frontier for treatment of neurological disorders

@article{Rinaldi2018AntisenseOT,
  title={Antisense oligonucleotides: the next frontier for treatment of neurological disorders},
  author={Carlo Rinaldi and Matthew J. A. Wood},
  journal={Nature Reviews Neurology},
  year={2018},
  volume={14},
  pages={9-21}
}
Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and modulate protein expression over two decades ago; however, progress translating these agents into the clinic has been hampered by inadequate target engagement, insufficient biological activity, and off-target toxic effects. Over the years, novel chemical modifications of ASOs have been employed to address these issues. These modifications, in combination with elucidation of the mechanism of action of ASOs… 

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