AAV Vector Retinal Target Gene Transfer Therapy

Publication: eyehopefoundation.org | Publication Date: February 14, 2020

Authors: Professor Hamel

Abstract

A reliable mouse model of Wolfram syndrome with the visual degradation has been developed. Initial tests with AAV-drive gene therapy has shown promising results in those mice to partially rescue the vision of wolfram syndrome patients. In this supported project, professor Hamels team will investigate higher dose concentrations of AAV-based gene therapy on mouse level. If it is successful, the group will proceed to pave the way towards a treatment for the vision problem of Wolfram-patients.