Developing Stem Cell Therapies for Cystic Fibrosis
Cystic fibrosis (CF) remains the most lethal autosomal recessive disorder in Caucasians, affecting around 70,000 people worldwide. Although multiple organs are affected, it is the pulmonary manifestations that are the primary cause of CF-related morbidity and mortality. Despite the identification of almost 2,000 disease-causing mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), the precise role of CFTR in the development and progression of CF lung disease is still poorly understood. This is largely due to a lack of in vivo/in vitro models that closely reflect human lung development and CF pathology. We are working on:
- the development of patient specific iPSC-based models and high throughput screening tools for CF;
- the generation of an expandable and transplantable population of lung stem cells from iPSC with the capacity for regeneration of an injured lung utilizing the ferret as a pre-clinical model.