Properties
Description
PTC124, soluble in DMSO and insoluble in ethanol and water, blocks the activity of the cystic fibrosis transmembrane conductance regulator protein, which plays a crucial role in the movement of chloride ions across cell membranes. It targets CFTR (HEK293 cells).
Molecular Formula
C15H9FN2O3
Targets
CFTR (HEK293 cells)
Solubility
DMSO: 55 mg/mL (193.49 mM); Water: Insoluble; Ethanol: Insoluble
Identity
Confirmed by NMR/HPLC/MS.
Applications
PTC124 is a CFTR inhibitor researched for its potential in treating genetic disorders such as cystic fibrosis by promoting ribosomal read-through of premature stop codons.