Dr. Catherine Argyriou has recently joined the faculty at Children’s Hospital Los Angeles (CHLA), where she aims to enhance the understanding and treatment of genetic diseases through innovative research. Dr. Argyriou, who holds a PhD, brings with her a wealth of experience and a passion for rare disease research that began during her academic career in Winnipeg, Manitoba, Canada.
As a researcher, Dr. Argyriou is particularly intrigued by the molecular mechanisms that influence human health. She states, “There is no limit to what we can observe or strive to understand, and there is no limit to using this information to achieve a desired effect—I think nothing is more exciting than contributing to that story.” This enthusiasm for scientific exploration guided her to pursue her doctorate and focus on the complexities of genetic disorders.
Focus on Gene Therapy for Rare Diseases
After spending 15 years at McGill University in Montréal, Québec, Canada, Dr. Argyriou felt compelled to move to the United States. CHLA’s Vision Center presented a unique opportunity for her to translate research discoveries into effective therapies. “The main goal of my research is to better understand the mechanisms of genetic diseases and to develop gene-based therapies to treat them,” she explains.
Dr. Argyriou’s work targets multi-systemic diseases that can affect various organs, including those that result in vision loss. One of her notable projects involves developing a retinal gene therapy for Zellweger Spectrum Disorder, a condition that disrupts the function of peroxisomes, cellular structures essential for various metabolic processes. In addition, she collaborates with colleagues at the Vision Center on precision medicine approaches that utilize gene correction techniques to combat inherited retinal degenerations.
This cutting-edge research leverages patient-derived models, including induced pluripotent stem cells and retinal organoids. Dr. Argyriou emphasizes that these models enable her team to test gene editing methods with the goal of preventing vision loss on an individualized basis. Such advancements could represent a significant shift in how therapies are developed for patients suffering from genetic disorders.
Building a New Research Lab
As she establishes her research lab at CHLA, Dr. Argyriou is eager to advance her projects and collaborate with her team. Her commitment to supporting patient communities is evident in her belief that “an unlucky event can bring people together in the patient communities and create these beautiful bonds and networks that we are privileged to support.”
Beyond her professional endeavors, Dr. Argyriou enjoys activities that keep her balanced, including ice skating, walking, reading, and ballet. Her diverse interests reflect a well-rounded individual dedicated not only to her patients but also to her personal well-being.
Dr. Argyriou’s arrival at The Saban Research Institute of CHLA marks an exciting chapter in her career and a promising development in the field of gene therapy and rare disease research. Her work is anticipated to contribute significantly to both scientific knowledge and patient care.
