VRT-325 is a small molecule designed to target the cystic fibrosis transmembrane conductance regulator (CFTR), specifically addressing the ΔF508 mutation associated with cystic fibrosis. This compound promotes the efflux of ΔF508-CFTR from the endoplasmic reticulum, thereby restoring chloride transport in epithelial cells derived from CF patients. VRT-325 is valuable for research into therapeutic strategies aimed at correcting CFTR function and enhancing epithelial ion transport in cystic fibrosis.
VRT-325 is a small molecule designed to target the cystic fibrosis transmembrane conductance regulator (CFTR), specifically addressing the ΔF508 mutation associated with cystic fibrosis. This compound promotes the efflux of ΔF508-CFTR from the endoplasmic reticulum, thereby restoring chloride transport in epithelial cells derived from CF patients. VRT-325 is valuable for research into therapeutic strategies aimed at correcting CFTR function and enhancing epithelial ion transport in cystic fibrosis.
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