Current Research Description

Our lab focuses on neuromuscular disorders such as Duchenne Muscular dystrophy, which is a devastating disease leading to death of patients at 20-30 years of age. Our lab has developed an expertise in translational research, an emerging field which can be summarized as understanding the cause of a disease and learning from it in order to develop innovative therapeutic strategies. We are fully committed to continuing to integrate basic science and gene therapy skills in order to advance understanding and treatment of the muscular dystrophies. Our long-term projects focus on RNA and DNA editing and gene transfer as therapies for neuromuscular disorders, but they will also include more fundamental research on muscle function. In particular, our lab uses the newest technologies, such as virus-mediated exon-skipping and the CRISPR/Cas9 genome editing tool.