Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction

Neurotherapeutics. 2019 Apr;16(2):348-359. doi: 10.1007/s13311-019-00729-0.


Antisense oligonucleotides (ASOs) have shown potential as therapeutic molecules for the treatment of inner ear dysfunction. The peripheral sensory organs responsible for both hearing and equilibrium are housed within the inner ear. Hearing loss and vestibular balance problems affect a large portion of the population and limited treatment options exist. Targeting ASOs to the inner ear as a therapeutic strategy has unique pharmacokinetic and drug delivery opportunities and challenges. Here, we review ASO technology, delivery, disease targets, and other key considerations for development of this therapeutic approach.

Keywords: RNA interference; Usher syndrome; antisense oligonucleotides; deafness; hearing loss; sensorineural hearing loss; splicing.

Publication types

  • Review

MeSH terms

  • Animals
  • Ear, Inner
  • Hearing Loss, Sensorineural / drug therapy*
  • Humans
  • Molecular Targeted Therapy
  • Oligonucleotides, Antisense / therapeutic use*
  • Usher Syndromes / drug therapy*


  • Oligonucleotides, Antisense