Our laboratory studies the control of breathing and pulmonary mechanics in neuromuscular disorders, including Pompe disease, Duchenne muscular dystrophy, spinocerebellar ataxia, and amyotrophic lateral sclerosis. We investigate the extent of respiratory pathophysiology and neuromodulation that contribute to respiratory dysfunction using humanized murine models. We also assess the effects of next-generation muscle- and CNS-tropic AAV gene therapies on both respiratory and systemic pathology.
Mission StatementÂ
Our overarching goal is to positively impact the lives of patients with neuromuscular diseases through scientific discovery. Our specific research focus is to elucidate mechanisms that contribute to respiratory insufficiency in neuromuscular disorders and to explore novel therapeutics to treat this pathology.
