�鶹��

Jakob Haldrup's vision is a future where new-borns with inherited genetic diseases can be treated by a programmable ´genome editing´ agent, guided to fix the genetic error like a ‘genomic GPS’. The main challenge lies in delivering the 'genome editing' toolkits without triggering an immune response. Additionally, using a delivery system that vanishes after correcting the genetic defect is crucial; otherwise, it might lead to new complications. In this project, I combine the potency of computational biology with cutting-edge molecular techniques to repurpose human proteins for delivering 'genome editing' toolkits. As the human body is already familiar with these vehicles, they can circulate and deliver their cargo without being neutralized, thereby enhancing both potency and safety. As proof-of-concept, I will target neurodegenerative diseases in the central nervous system.