Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction.
Neurotherapeutics
; 16(2): 348-359, 2019 04.
Article
em En
| MEDLINE
| ID: mdl-30972560
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.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos Antissenso
/
Síndromes de Usher
/
Perda Auditiva Neurossensorial
Limite:
Animals
/
Humans
Idioma:
En
Revista:
Neurotherapeutics
Ano de publicação:
2019
Tipo de documento:
Article