Discovery of CFTR modulators for the treatment of cystic fibrosis.
Miquéias Lopes-PachecoNicoletta PedemonteGuido VeitPublished in: Expert opinion on drug discovery (2021)
Highly effective CFTR modulator drugs are expected to deeply modify the disease course for the majority of individuals with CF. A multitude of experimental approaches have been established to accelerate the development of novel modulators. CF patient-derived specimens are valuable cell models to predict therapeutic effectiveness of existing (and novel) modulators in a precision medicine approach.