Article
High-Content Surface and Total Expression siRNA Kinase Library Screen with VX-809 Treatment Reveals Kinase Targets that Enhance F508del-CFTR Rescue.
Molecular pharmaceutics - 5 Mar 2018
Perkins Lydia A, Fisher Gregory W, Naganbabu Matharishwan, Schmidt Brigitte F, Mun Frederick, Bruchez Marcel P
Abstract excerpt
The most promising F508del-CFTR corrector, VX-809, has been unsuccessful as an effective, stand-alone treatment for CF patients, but the rescue effect in combination with other drugs may confer an acceptable level of therapeutic benefit. Targeting cellular factors that modify trafficking may act to enhance the cell surface density of F508-CFTR with VX-809 correction. Our goal is to identify druggable kinases that...
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