Article
Characterization of the mechanism of action of RDR01752, a novel corrector of F508del-CFTR.
Biochemical pharmacology - 1 Oct 2020
Lopes-Pacheco Miquéias, Silva Iris A L, Turner Mark J, Carlile Graeme W, Sondo Elvira, Thomas David Y, Pedemonte Nicoletta, Hanrahan John W, Amaral Margarida D
Abstract excerpt
Despite progress in developing pharmacotherapies to rescue F508del-CFTR, the most prevalent Cystic Fibrosis (CF)-causing mutation, individuals homozygous for this mutation still face several disease-related symptoms. Thus, more potent compound combinations are still needed. Here, we investigated the mechanism of action (MoA) of RDR01752, a novel F508del-CFTR trafficking corrector. F508del-CFTR correction by...
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