Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction

Michelle L. Hastings, Timothy A. Jones

Research output: Contribution to journalReview article

4 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)348-359
Number of pages12
JournalNeurotherapeutics
Volume16
Issue number2
DOIs
StatePublished - Apr 15 2019

Keywords

  • RNA interference
  • Usher syndrome
  • antisense oligonucleotides
  • deafness
  • hearing loss
  • sensorineural hearing loss
  • splicing

ASJC Scopus subject areas

  • Pharmacology
  • Clinical Neurology
  • Pharmacology (medical)

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