LGMD “Spotlight Interview on Research”
Affiliation: University of Minnesota
Role or Position: Professor of Medicine
What education and training did you have to arrive at your current position?
PhD in Physiology at the University of Campinas (UNICAMP) in Sao Paulo, Brazil, followed by postdoctoral studies at the Whitehead Institute at MIT, Cambridge-MA.
What led you to follow a career in research and in studying muscular dystrophy in particular?
I wanted to be a scientist since I was a little kid. Throughout my career, the main motivation has been the desire to make in impact in people’s lives through our research.
What topics are you studying?
The main topic of research in our laboratory is the development of stem cell-based therapies for muscular dystrophies, and this includes a better understanding of the molecular signature of pluripotent stem cell-derived myogenic progenitors and engrafted stem cells, the signals that allow for these cells to home to diseased muscles upon systemic transplantation and promote muscle regeneration, gene correction of LGMD patient-specific induced pluripotent stem (iPS) cells for assessing the potential of an autologous stem cell-based therapy, and optimization studies for the scalability, manufacturing and safety of pluripotent stem cell-derived myogenic progenitors for therapeutic applications.
How will your work help patients? Is it more scientific in nature or might it become a treatment for LGMDs or MDs in general?
When we began these studies in 2004, they were purely basic science, but as we advanced our research over the years, we feel we are now much closer to translating this approach into a potential treatment for LGMD and other MDs. We had a pre-IND meeting with the FDA in 2017 to align our short- and long-term goals.
What would you like patients and others interested in LGMD to know about research (your own projects and about the field in general)?
There are many obstacles and many unknowns in the road of bringing a new therapeutic treatment for MD testing, but our lab and others are working fearlessly towards this goal.
What inspires you to continue working in this field?
The possibility that our work may make a difference in the lives of patients with MDs.
How can patients encourage you and help your work?
The strength and hope of MD patients constantly give us significant encouragement. Over the years, patients have helped our work through tissue donation and financial support and we are grateful for their support.
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* * * To learn more about Limb Girdle Muscular Dystrophy (LGMD), please visit our website at: https://lgmd-info.org