Viability of long-term gene therapy in the cochlea

dc.contributor.authorAtkinson, Patrick
dc.contributor.authorWise, Andrew
dc.contributor.authorFlynn, Brianna
dc.contributor.authorNayagam, Bryony
dc.contributor.authorRichardson, Rachael
dc.date.accessioned2014-04-27T23:39:29Z
dc.date.available2014-04-27T23:39:29Z
dc.date.issued2014-04-22
dc.description.abstractGene therapy has been investigated as a way to introduce a variety of genes to treat neurological disorders. An important clinical consideration is its long-term effectiveness. This research aims to study the long-term expression and effectiveness of gene therapy in promoting spiral ganglion neuron survival after deafness. Adenoviral vectors modified to express brain derived neurotrophic factor or neurotrophin-3 were unilaterally injected into the guinea pig cochlea one week post ototoxic deafening. After six months, persistence of gene expression and significantly greater neuronal survival in neurotrophin-treated cochleae compared to the contralateral cochleae were observed. The long-term gene expression observed indicates that gene therapy is potentially viable; however the degeneration of the transduced cells as a result of the original ototoxic insult may limit clinical effectiveness. With further research aimed at transducing stable cochlear cells, gene therapy may be an efficacious way to introduce neurotrophins to promote neuronal survival after hearing loss.en_US
dc.description.sponsorshipThe Garnett Passe and Rodney Williams Memorial Foundation, Action on Hearing Loss, The University of Melbourne - Department of Otolaryngology, The Royal Victorian Eye and Ear Hospital, NHMRC (GNT1024350)en_US
dc.identifier.citationAtkinson, P. J., Wise, A. K., Flynn, B. O., Nayagam, B. A. & Richardson, R. T. (2014). Viability of long-term gene therapy in the cochlea. Scientific Reports 4, article no. 4733.en_US
dc.identifier.urihttp://repository.bionicsinstitute.org:8080/handle/123456789/78
dc.language.isoenen_US
dc.publisherNature Publishing Groupen_US
dc.subjectGene Therapyen_US
dc.subjectDeafnessen_US
dc.subjectNeurotrophinsen_US
dc.titleViability of long-term gene therapy in the cochleaen_US
dc.typeArticleen_US
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