To lay the foundation for the Path to a Cure, an ambitious initiative to accelerate treatments for the underlying cause of the disease and a cure for cystic fibrosis, we need to understand the cells to target and the challenges of delivering genetic-based therapies (such as gene editing and gene therapy).
In this plenary, Brian Davis, PhD from the University of Texas Health Science Center at Houston, will focus on the importance of understanding the underlying cellular architecture and the biology of the epithelial tissues (e.g., the lungs) that must be treated. Maria Limberis, PhD from the University of Pennsylvania, will explore the complex physical properties of the lungs that make the surface of the airway an effective barrier against viral and bacterial pathogens, but that also make the successful delivery of genetic-based therapies more difficult.
Their presentations will provide insight into the current status of promising state-of-the-art genetic-based therapies as well as put in context a common vocabulary and conceptual framework as we move forward with developing a cure for all.
Originally recorded October 21, 2020.