AAV-Mediated Gene Delivery to the Inner Ear.
Methods Mol Biol
; 1950: 271-282, 2019.
Article
em En
| MEDLINE
| ID: mdl-30783980
ABSTRACT
Cochlear gene therapy has made tremendous strides over the past 5 years. The first study documenting successful restoration of congenital hearing loss using AAV-mediated gene therapy occurred in a mouse model of deafness lacking vesicular glutamate transporter 3 (VGLUT 3). This study utilized a trans-bulla round window membrane (RWM) delivery approach. Since this study, these methodologies have been applied to a number of other mouse models of genetic deafness with varying degrees of success, lending promise for future clinical application of this burgeoning technology. Here we describe a method of virally mediated gene delivery into the cochlear scala tympani through the RWM. This method involves negligible damage to essential structures of the middle and inner ear while preserving hearing. The efficacy of this surgical technique will be demonstrated by the restoration of hearing to the VGLUT3 knockout mice (a mouse model of congenital deafness) after delivery of VGLUT3 gene to the inner ear using an adeno-associated virus as a vector.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Técnicas de Transferência de Genes
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Dependovirus
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Vetores Genéticos
/
Orelha Interna
Limite:
Animals
Idioma:
En
Revista:
Methods Mol Biol
Assunto da revista:
BIOLOGIA MOLECULAR
Ano de publicação:
2019
Tipo de documento:
Article
País de afiliação:
Estados Unidos