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The goal of EditYR is to improve the efficiency, safety, and durability of therapies for treatment of genetic diseases - conditions caused by inherited errors in genetic code.

Current advanced gene therapies are based on delivering “healthy” copies of defective genes into cells. However, these copies can be lost over time or inserted randomly into the genome, potentially leading to additional health complications. A safer and more permanent solution lies in molecular tools that can target carefully selected, safe sites in the cell`s genetic material, and stably insert a healthy gene copy at these locations.

The project will focus on microbial enzymes known as tyrosine recombinases. These enzymes act as both glue and scissors – a single enzyme can cut the genetic material and insert a new segment with high precision, without introducing additional errors. However, directing tyrosine recombinases to arbitrary locations is not straightforward. The main objective of the project is to develop new strategies for their precise targeting to selected genomic locations. This will be achieved through a combination of comparative sequence analysis, structural modelling, and high-throughput experimental approaches. Because safety is of utmost importance for any therapeutic application, we will identify, test, and rigorously select sites where insertion of new genes does not disrupt normal cellular function.