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Systems Approaches to Unravel Molecular Function: High-content siRNA Screen Identifies TMEM16A Traffic Regulators as Potential Drug Targets for Cystic Fibrosis

Pinto, Madalena C. ; Botelho, Hugo M. ; Silva, Iris A.L. ; Railean, Violeta ; Neumann, Beate ; Pepperkok, Rainer ; Schreiber, Rainer ; Kunzelmann, Karl ; Amaral, Margarida D.



Abstract

An attractive approach to treat people with Cystic Fibrosis (CF), a life-shortening disease caused by mutant CFTR, is to compensate for the absence of this chloride/bicarbonate channel by activating alter-native (non-CFTR) chloride channels. One obvious target for such "mutation-agnostic" therapeutic approach is TMEM16A (anoctamin-1/ANO1), a calcium-activated chloride channel (CaCC) which is also ...

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