I am focus on bringing Gene Editing to help translational research in Pompe and other rare motor and neurodegenerative disorders to develop actual curative therapies. Some of my current research topics include: -Gene Therapy -Pompe Disease -Viral Vectors -Safe and Specific Promoter Engineering -Gene Editing -Disease Modeling -Preclinical testing and characterization of novel therapeutics -Improvement and repurpose of advance therapeutic tools