CRISPR Therapeutics
We are developing next-generation CRISPR-based genetic medicines to treat severe medical conditions with a main focus on neurological and metabolic diseases. Unlike conventional therapies, genome editing holds the potential for long-lasting therapeutic effects. More broadly, our work demonstrates how the versatility of CRISPR enzymes can be harnessed to create precise genetic edits toward once-and-done treatments.
Neuromuscular Diseases
Neuromuscular diseases are a major burden on humanity. Our research focuses on spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, and amyotrophic lateral sclerosis (ALS), a severe neurodegenerative disease. With no transformative treatments currently available for sporadic ALS, and over 30,000 affected individuals in the U.S. alone, innovation is urgently needed. We investigate the molecular drivers of these diseases to develop innovative therapies and identify biomarkers for early diagnosis and disease monitoring.
Applied Physiology
A longstanding focus of our research is understanding muscle fiber remodeling. We have demonstrated that oxidative metabolism plays a crucial role in protecting against muscle loss, laying the groundwork for our ongoing studies. We are also interested in the pleiotropic effects of physical exercise as a non-pharmacological therapy for multiple conditions. These efforts intersect with our other research areas, creating an opportunity to integrate molecular approaches within the broader context of human physiology.
