This workshop highlights advancements in new and emerging technologies progressing towards a cure. Discussions will include development of tools/strategies focused on rare and nonsense CFTR variants with translatable relevance to all CFTR mutations.
Summarize molecular defects attributable to CFTR premature truncation codons, including the role of nonsense mediated decay and its potential as a therapeutic target.
Compare approaches for CFTR gene repair or replacement in primary airway epithelia.
Evaluate state-of-the-art directed differentiation of airway progenitor cells from pluripotent source material.