Xinyi Chen

Finding cures for monogenic diseases is an area of urgent unmet medical need. The rapid advances in gene editing technology provided promising solutions, but their translation to the clinic is largely hindered by the lack of a safe and efficacious delivery vehicle. Currently existing delivery vehicles such as viral particles or nanoparticles suffer from payload size limitation, non-specificity, and lack of external control. To address these limitations, I propose using genetically engineered cells, which have been used for cancer therapy, to accomplish the delivery task in the patient body instead. The delivery is achieved through repurposing the natural cell-cell communication and material exchange pathways. Just like writing computer programs, I aim to compose “genetic programs” that instruct engineered cells to produce gene editing tools, precisely target the diseased cells, and efficiently deliver the gene editing tools to them at the right time and with minimal side effects.