Gene therapies and models for neurodegenerative diseases

We are interested in developing this alternative gene therapeutic strategy relying on the delivery of neuro-protective or anti-apoptotic genes. This mode of gene therapy equally relies on our ability to efficiently manipulate the AAV-based vectors to target various cell types in the retina and we have created a number of AAV serotypes with desirable properties in this context.

Research areas

• Development of AAV-based gene therapies for retinal diseases.
• Animal models of human neurodegenerative disease.
• Development of new AAV vectors for gene delivery to the retina.
• Understanding the changes in viral distribution in retinas undergoing disease