Usher Syndrome in the Inner Ear: Etiologies and Advances in Gene Therapy.
Int J Mol Sci
; 22(8)2021 Apr 10.
Article
en En
| MEDLINE
| ID: mdl-33920085
ABSTRACT
Hearing loss is the most common sensory disorder with ~466 million people worldwide affected, representing about 5% of the population. A substantial portion of hearing loss is genetic. Hearing loss can either be non-syndromic, if hearing loss is the only clinical manifestation, or syndromic, if the hearing loss is accompanied by a collage of other clinical manifestations. Usher syndrome is a syndromic form of genetic hearing loss that is accompanied by impaired vision associated with retinitis pigmentosa and, in many cases, vestibular dysfunction. It is the most common cause of deaf-blindness. Currently cochlear implantation or hearing aids are the only treatments for Usher-related hearing loss. However, gene therapy has shown promise in treating Usher-related retinitis pigmentosa. Here we review how the etiologies of Usher-related hearing loss make it a good candidate for gene therapy and discuss how various forms of gene therapy could be applied to Usher-related hearing loss.
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Terapia Genética
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Retinitis Pigmentosa
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Síndromes de Usher
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Pérdida Auditiva
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Oído Interno
Tipo de estudio:
Etiology_studies
Límite:
Humans
Idioma:
En
Revista:
Int J Mol Sci
Año:
2021
Tipo del documento:
Article
País de afiliación:
Estados Unidos