Gene therapy for LCA and Age-Related Macular Degeneration
The aim of this project was to investigate the impact of gene therapy for Leber Congenital Amaurosis (LCA) using a dog model of the disease.
Project Results and Impact
Following gene therapy, the vision impaired dogs showed remarkable improvement in their sight. After this success, the team focused its attention on Age Related Macular Degeneration (AMD).
Professor Rakoczy and her team developed a promising approach called Secretion Gene Therapy to treat wet AMD. Their preclinical trials have shown positive results in inhibiting the growth of harmful blood vessels and maintaining vision. They are now seeking approval to conduct human trials in Australia, with the aim of starting clinical trials soon.