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Discovery of CFTR modulators for the treatment of cystic fibrosis.

Miquéias Lopes-PachecoNicoletta PedemonteGuido Veit
Published 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.
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