Sorscher R01 “Rare Mutations in Cystic Fibrosis: Overcoming Barriers to Personalized Medicine”
Eric Sorscher awarded R01 titled "Rare Mutations in Cystic Fibrosis: Overcoming Barriers to Personalized Medicine"
The project will investigate very rare CFTR variants in model systems such as pluripotent stem cells differentiated to an airway epithelial phenotype. We will also advance new recombinant cell lines for studies of CF disease mechanism. A clinical trial will be conducted to test leading edge CF drugs in rare forms of the disease – as a means to develop new tools for “precision” CF therapy and help promote modulator access.