James Novak, Ph.D.'s research team at Children's National Hospital is focused on the development of therapeutic strategies for muscular dystrophies that target the underlying genetic cause of the disease and ameliorate disease severity and progression. To date, a major clinical challenge has been to improve targeted drug delivery and retention within skeletal and cardiac muscle tissue to increase drug efficacy. Towards this challenge, a major focus of our team has been to identify how the complex pathological processes in DMD, and other muscular dystrophies, modulate muscle-specific drug delivery and therapeutic efficacy of various gene therapies to develop novel delivery strategies to improve drug pharmacokinetics and clinical benefit. In addition, his team is focused on unravelling the complex and aberrant cellular and molecular processes that contribute to the onset and rapid progression of muscular dystrophies to develop therapeutic targets for clinical translation.
