Inherited retinal diseases are characterized by dysfunction and degeneration of the photoreceptors and/or the retinal pigment epithelium, leading to progressive vision loss and blindness. After winning a grant of just over DKK 3 million (EUR 400,000) from the Velux Foundation, PhD student Silja Hansen MD aims to develop a new treatment based on prime editing to slow the progression of inherited retinal diseases. This editing has the potential to correct all kinds of point mutations as well as smaller deletions and insertions that cover many of the variants underlying inherited retinal diseases. This tool functions as an all-in-one system thanks to the pegRNA that directs the enzyme complex to the target site and as its own template because it does not require a double-strand break. One of the challenges of prime editing is to find a pegRNA that can create the desired editing and achieve high targeting efficiency. Another major challenge is the delivery of the gene editing cargo. Previous studies have explored the possibility of using a dual adeno-associated virus to provide a split master editor system. They would like to test this strategy but would also like to investigate different delivery options, including non-viral delivery methods.
