Dhindsa JS et al, Motor axonopathies in a mouse model of Duchenne muscular dystrophy. Sci Rep. 2020 Jun 2
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease with no current cure. Boys with DMD have progressive respiratory weakness and many have neurological impairment. In our laboratory, we examine the impact of DMD on breathing and neurological pathology in a novel humanized mouse model of DMD that has the human genetic mutation. We also use novel adeno-associated viral (AAV) gene therapy to treat the respiratory and neuropathology in DMD.
