During this award, a novel gene editing therapy was further developed and tested. This therapy uses molecular scissors to permanently remove mutations for 50% of Duchenne muscular dystrophy patients, a devastating muscle wasting disease that leads to premature death. Throughout this project we assessed the best dose level, safety, and manufacturing of the therapy. We also conducted a pre-IND meeting with FDA to discuss our data and future plans. We obtained a clear path to advance our therapy to human testing in the next few years.