Rachelle Crosbie-Watson, professor and vice-chair of graduate education at the University of California Los Angeles, was awarded an MDA research grant this summer totaling $300,000 over a period of three years to test new therapies for cardiomyopathy (heart muscle weakness) associated with Duchenne muscular dystrophy (DMD).
Can you tell us a little bit about your research project?
The research project is focused on testing new therapies for cardiomyopathy associated with DMD, using mouse models that display severe forms of cardiac and skeletal muscle disease. We hope to determine whether restoring a protein called utrophin to DMD cardiomyocytes (heart muscle cells) will prevent membrane damage, degeneration, and cardiac failure.
Is this your first MDA grant?
It’s actually not! In fact, my first MDA award was the Robert G. Sampson Neuromuscular Disease Research Award (1996) that supported my postdoctoral research in the laboratory of Dr. Kevin P. Campbell (HHMI/ University of Iowa), which was transformative in guiding my future career in muscular dystrophy.
Research grants from MDA are very impactful in driving research forward to improve basic and translational research. Support from the MDA has been a cornerstone in our research on membrane stabilizers for Duchenne muscular dystrophy.
What inspired you to study DMD?
I have been working on DMD since my postdoctoral fellow with Dr. Kevin P. Campbell. We continue to be inspired to investigate basic molecular mechanisms and therapeutic targets as part of our connection to the DMD community through the UCLA Center for Duchenne Muscular Dystrophy.
What is your area of focus and why is it important?
Our research is focused on new targets that stabilize the cell membrane, which is very fragile in Duchenne skeletal and cardiac muscles. We hypothesize that these stabilizers may prevent the muscle cell from becoming injured during normal use.
Addressing the primary cause of disease, which is membrane instability and loss of muscle cell contact with its surrounding matrix, has the potential to address skeletal muscle, respiratory, and cardiac aspects of disease.
Why is it important for MDA to continue to fund DMD research?
For the first time, there are numerous ongoing clinical trials focused on DMD. This is a critical and important time in Duchenne-focused research, and now is the time to drive these new ideas and experimental treatments to become real treatments.
What do you feel people impacted by DMD can have the most hope about with research right now?
The hope that all of us feel now is derived from the advancements in preclinical treatments that have led to worldwide clinical trials for Duchenne. These clinical trials are leading the way for future treatments and second-generation compounds.
See more about the impact we’re making this year for DMD and all types of muscular dystrophy.