Supervisor: A/Prof Guei-Sheung Liu

Email: gsliu@unimelb.edu.au

Suitable for: PhD

Many genetic eye diseases resulting in blindness manifest as degeneration of retinal cells and are collectively termed inherited retinal diseases (IRDs). IRDs are the most common cause of blindness in working-age Australians. Although the specific mutations, and its respective gene, that cause IRDs are mostly known, these conditions remain largely untreatable.

Here, the aim of the project is to develop a generalisable gene therapy for IRDs through a new gene editing tool, “RNA base editing”. The majority of IRDs are caused by single-base mutations that impair protein expression or function resulting in disease progression. RNA base editing is designed to correct single base mutations in RNA transcripts, the messenger of the genes used to make proteins, and restore functional protein(s).

This proposal is built on a recently developed CRISPR/Cas RNA base editor, which is compact in size and can be packaged in a clinical applicable viral vector. We will employ this newly developed CRISPR/Cas RNA editor to enable direct correction of pathogenic IRD causing single base variants.