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Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction.

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.

SUBMITTER: Hastings ML

PROVIDER: S-EPMC6554385 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature

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Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction.

Hastings Michelle L ML Jones Timothy A TA

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 20190401 2

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 rev ...[more]

PMID: 30972560

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