What do you love most about your work?
I love having a clear focus on patients. We are not just studying biology in theory. We are working to correct the root cause of devastating genetic diseases and restore normal tissues in the same individual who donated the cells. The science, like gene editing and stem cell biology, is not an endpoint. It is a bridge. When done rigorously and safely, it can change a patient's life from debilitating to functional.
How has cell and gene therapy revolutionized your field?
Cell and gene therapy enable us to address the root cause of disease, which has been transformational. In the past, we could only manage symptoms by providing wound care, pain relief, and supportive treatments for skin disorders like EB. Now, we can directly target the faulty gene responsible for the condition.
Advances in gene editing tools, safer delivery systems, and stem cell technologies, especially induced pluripotent stem cells (iPSCs), have made it possible to repair a patient’s own cells, grow healthy tissue in the laboratory, and prepare it for transplantation. These technological advances shift the focus from temporary management to potentially durable, long-term correction.
Tell us about your current research.
My research centers on EB, with a particular emphasis on the most severe type, recessive dystrophic EB, or RDEB. People born with EB have very fragile skin that blisters and tears easily because of a mistake in one of their genes. Right now, there is no cure, but we hope to change that.
In our lab, we take a small sample of a patient’s skin cells and reprogram them into iPSCs. These cells can turn into many different types of cells in the body. We fix the genetic mistake in these cells and then grow them into healthy new skin cells.
This work is highly collaborative. I work closely with Dennis Roop, PhD, and Igor Kogut, PhD, on the scientific development, and with clinicians such as Anna Bruckner, MD, and Emily Gorell, DO, who care for EB patients. Their expertise and partnership make it possible to advance this work from the laboratory toward real patient treatment.
How has Gates Institute helped you advance your work?
Gates Institute provides the infrastructure and expertise needed to translate our discoveries into clinical-grade therapies. At the Gates Biomanufacturing Facility, we can produce patient-specific stem cells and gene-corrected skin cells under the strict quality and safety standards required for human trials. This ensures that what we develop in the laboratory can move toward clinical trials.
In addition, the Institute has supported the regulatory pathway. Its guidance in preparing materials for FDA engagement has been critical in refining our manufacturing strategy, safety testing plans, and overall translational roadmap.
What does philanthropy mean to you?
Philanthropy, to me, means urgent partnership. It involves patients, families, and communities choosing to invest in science not just abstractly, but with hope, trust, and a deeply personal stake in the outcome. The support from benefactors enables us to accelerate progress, take scientific risks, and build the necessary infrastructure for translation. Philanthropy bridges the gap between discovery and treatment. It transforms compassion into momentum and helps bring real therapies closer to patients.
Non-profit organizations like EB Research Partnership, Cure EB, Epidermolysis Bullosa Medical Research Foundation, and DEBRA International also play a transformative role. They fund high-risk, high-impact research, connect scientists directly with patients, and build accountability around clear milestones.
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