Summary
PROJECT SUMMARY: Gene therapy (GT) for the hemophilias is poised to fulfill the promise of a safe, affordable, lifelong correction following a single treatment. Yet clinical trials employing AAV vectors to treat adult hemophilia A (HA) patients have underscored the risk of hepatoxicity and genotoxicity in adult patients receiving GT. In addition, the use of AAV-based GT is prevented by pre-existing AAV immunity in a high percentage of patients. While FVIII inhibitors have not yet been seen in GT trials, inhibitors usually appear within the first 50 exposure days to FVIII, and since all enrolle
