The project aims to improve cochlear implant performance via integrated gene therapy. A neurotrophin gene cassette will be delivered to cells adjacent to the electrode array using electrical pulses. This drives regeneration of the auditory nerve fibres and considerably improves cochlear implant performance. This study will optimize the therapeutic gene construct and cochlear implant –based gene delivery controller, and undertake an initial clinical trial to evaluate safety and efficacy.
Development Of Novel Gene Therapy Vectors For Thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$287,307.00
Summary
Thalassaemia, is a common inherited disorder affecting haemoglobin synthesis. Synthesis of ?/?-globin chain is balanced during normal red blood cell production. Any disruption in the ratio of ?/?-globin chain results in anaemia. In this study, we will explore gene therapy strategies to restore balanced ?:? globin expression and ultimately improve the severely anaemic phenotype in ?-thalassaemia patients.