History & Future of CFTR Modulators - Alan Verkman, MD, PhD - Audio

The first “potentiators” of mutant CFTRs, which rescue their defective chloride channel function, were identified by high-throughput screening and reported by Dr. Verkman’s lab in 2003, and in 2005 they reported the first “correctors” of defective cellular processing of the deltaF508 CFTR mutant. Dr. Verkman provides a comprehensive overview of the history and future of CFTR modulators, advances in “CFTR-agnostic” therapies, and other hopes for transformative CF therapeutic options.