Article
Correctors of the Major Cystic Fibrosis Mutant Interact through Membrane-Spanning Domains.
Molecular pharmacology - 1 Jun 2018
Laselva Onofrio, Molinski Steven, Casavola Valeria, Bear Christine E
Abstract excerpt
The most common cystic fibrosis causing mutation is deletion of phenylalanine at position 508 (F508del), a mutation that leads to protein misassembly with defective processing. Small molecule corrector compounds: VX-809 or Corr-4a (C4) partially restores processing of the major mutant. These two prototypical corrector compounds cause an additive effect on F508del/cystic fibrosis transmembrane conductance...
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