Translating in vitro CFTR rescue into small molecule correctors for cystic fibrosis using the Library of Integrated Network‐based Cellular Signatures drug discovery platform

• Main Authors: Matthew D. Strub (Author), Shyam Ramachandran (Author), Dmitri Y. Boudko (Author), Ella A. Meleshkevitch (Author), Alejandro A. Pezzulo (Author), Aravind Subramanian (Author), Arthur Liberzon (Author), Robert J. Bridges (Author), Paul B. McCray Jr (Author)
• Format: Book
• Published: Wiley, 2022-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The common ΔF508‐CFTR mutation results in protein misfolding and proteasomal degradation. If ΔF508‐CFTR trafficks to the cell surface, its...