Project Title: “Correction of a common Duchenne muscular dystrophy mutation by homology-independent targeted integration (HITI)”
Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy, affecting approximately 1 in 5,000 boys, and has a high mortality rate in young adulthood. The disorder is caused by a genetic mutation, and there is currently no treatment that can reverse or halt progression of the disease.
Dr. Nicolau’s project will test whether homology-independent targeted integration (HITI) can be used to correct the most common mutation in the DMD gene. This research may result in an effective treatment for DMD that can stop or even reverse the progression of the disease.
Dr. Nicolau is a fellow in gene therapy at Nationwide Children’s Hospital.
This research is funded by the Muscular Dystrophy Association through the American Brain Foundation, in collaboration with the American Academy of Neurology.