APPTT
PTAC/CFTR
Amy Wong, PhD
University of Toronto & The Hospital for Sick Children
Juan Ianowski, PhD
University of Saskatchewan
Over the last few years there has been a renewed interest in studying the complexities of the cell biology of the airway epithelium and the contribution of the different cell types to CF lung disease pathobiology. A major rationale to pursue this research has been the need to identify the cellular targets for novel molecular therapies, such as gene therapy and gene editing.
Now it is evident that the airway consists of more than 10 different cell types, including some rare populations that express CFTR at high levels (e.g. ionocytes). Moreover, these cells are organized in complex structures, such as hillocks, with specific functions.
In this symposium we will discuss the complex cellular “ecology “ of the airway and how that complexity affects the development of molecular therapies to restore CFTR expression.
This symposium will generate a discussion on how the diversity of cell types in the airway epithelium, with different levels of CFTR expression, affects the development of molecular therapies to restore CFTR expression (e.g. gene therapy and/or editing). The focus is to understand and determine the who? (which cell should be targeted for gene therapy?), the what? (what are consequences of over expressing CFTR in the “wrong” cell type?), and the how? (how will these genetic editors be delivered for optimal targeting?).
Speaker: Ya-Wen Chen
Speaker: Ruobing Wang – Boston Children's Hospital
Speaker: Paul B. McCray, Jr., MD – University of Iowa
Speaker: Daniel Siegwart