Research Project 1
Therapies for currently untreatable autosomal recessive IRDs
Chief Investigator – Professor Robyn Jamieson, Children’s Medical Research Institute, Sydney Children’s Hospitals Network, Save Sight Institute – University of Sydney
Project Aim
- This project focuses on developing gene replacement therapies for autosomal recessive (AR) inherited retinal diseases (IRDs) where no current treatments exist.
- It will target genes small enough for AAV-mediated therapy, which has shown promise in previous successes like RPE65 gene therapy.
Research Project 2
Advancing Usher syndrome type 1B gene therapy with split intein
Chief Investigator – Dr Jiang-Hui (Sloan) Wang, Centre for Eye Research Australia
Project Aim
- This study is exploring a new treatment approach for Usher syndrome using a method that joins split proteins efficiently.
- It aims to deliver the full MYO7A protein in a mouse model of USH1B through a less invasive injection into the eye, which could lead to better treatment outcomes.
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