Canine factor VII deficiency as a test-platform to evaluate FVII gene therapy in humans

The review of our article titled “Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII” (1) by Drs. Bernardi and Pinotti (2) condenses basic biochemical and clinical data involving coagulation Factor VII. The authors highlight the physiological role played by tissue factor complexed with factor VII (FVII) or activated FVII in the initiation of coagulation and their involvement in pathological states such as thrombosis and cancer. They also provide very insightful views on human FVII deficiency and its specifics relative to other bleeding defects. Importantly, they discuss the concept of gene therapy for human monogenic coagulation defects. Within this framework, they present their comments on our article that demonstrated long-term expression of FVII following gene transfer in a large animal model of FVII deficiency.