Viability of long-term gene therapy in the cochlea

PJ Atkinson; AK Wise; BO Flynn; BA Nayagam; RT Richardson

Journal article

Viability of long-term gene therapy in the cochlea

PJ Atkinson, AK Wise, BO Flynn, BA Nayagam, RT Richardson

Scientific Reports | NATURE PORTFOLIO | Published : 2014

DOI: 10.1038/srep04733

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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 g..

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University of Melbourne Researchers

Andrew Wise Author

Bryony Nayagam Author

Rachael Richardson Author

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Grants

Awarded by National Health and Medical Research Council

Funding Acknowledgements

The authors would like to acknowledge and thank Assistant professor Clifford Hume from the University of Washington for the adenovirus used in this paper. The authors would also like to acknowledge the funding support from The 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, National Health and Medical Research Council (GNT1024350). The authors would like to acknowledge the support from the State Government of Victoria's Operational Infrastructure Support Program.