Cystic fibrosis cell models for high-throughput analysis and drug screening.

Abstract excerpt

Cystic fibrosis (CF) is a single-gene disorder that affects the lung, digestive system, and other organs. Mutations in the CF transmembrane conductance regulator (CFTR) gene are classified into several classes based on their pathogenic mechanism and clinical severity. The distinct and heterogeneous clinical behavior of each CF class and the respective CFTR mutations have made the development of a durable therapy...

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• Aminophenols
• Benzodioxoles
• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Drug Evaluation, Preclinical