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Sci Rep. 2014 Apr 22;4:4733. doi: 10.1038/srep04733.

Viability of long-term gene therapy in the cochlea.

Author information

1
1] Bionics Institute, East Melbourne, Victoria, Australia [2] Department of Otolaryngology, University of Melbourne, East Melbourne, Victoria, Australia.
2
1] Bionics Institute, East Melbourne, Victoria, Australia [2] Department of Otolaryngology, University of Melbourne, East Melbourne, Victoria, Australia [3] Department of Medical Bionics, University of Melbourne, East Melbourne, Victoria, Australia.
3
Bionics Institute, East Melbourne, Victoria, Australia.
4
1] Bionics Institute, East Melbourne, Victoria, Australia [2] Department of Audiology and Speech Pathology, University of Melbourne, Parkville, Victoria, Australia.

Abstract

Gene 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.

PMID:
24751795
PMCID:
PMC3994438
DOI:
10.1038/srep04733
[Indexed for MEDLINE]
Free PMC Article
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