Funding outcome

Duchenne muscular dystrophy (DMD) is a disease that mostly affects boys. It is caused by a change in a gene that makes a protein called dystrophin disappear. Dystrophin helps keep muscles strong and healthy, but when it is gone, muscles and the heart weaken over time, which can lead to early complications and a shorter life expectancy. There are treatments available that use artificial DNA-like molecules called antisense oligos (ASO), but they do not work as well as doctors would like, especially when it comes to restoring dystrophin in the heart. This research project is trying to find a new way to make the treatment work better by using ASO and adding a special peptide called DG9 developed by Dr. Yokota's research group. The goal is to make the treatment get into heart cells better. I will test the treatment in a mouse model of DMD by giving the medicine through a blood vessel and then doing tests to see how well it works. I will use tests to check muscle function, see if the ASO is getting into the cells better, and check the levels of certain proteins and RNAs in the mice. I expect that by giving the mice this type of medicine, the ASO will get into the heart and muscle cells better, and the mice will be able to use their muscles better with better-protected heart muscles.