Funding outcome

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder affecting 1 in every 5000 boys worldwide. Patients start showing symptoms around the age of 4 and gradually start losing muscle functions. Most children are unable to walk by the age of 12 and their life expectancy is around mid-twenties. There is no cure for this disease. DMD is caused by a mutation in the gene for the protein dystrophin. Dystrophin is important for supporting the muscle membrane. Without dystrophin, muscles are unable to function or repair themselves properly. This ultimately results in the loss of function in the arms, legs, and spine followed by breathing and heart problems. My focus of this study is to work on a treatment strategy for DMD using a novel technique called exon skipping. Here, I will use small DNA-like molecules along with a newly discovered delivery method to correct the genetic errors in DMD model mice. This will in turn restore their muscle function including the heart and improve life expectancy. I aim to formulate a treatment that can help about 20% of the patients with DMD. This study will provide a significant positive impact on the lives of children affected with DMD and allow them to live a life without the constraints of disability.