compound introduction
突变的G551D CFTR校正器。VRT-532通过增强突变体的ATP亲和力介导G551D-CFTR。VRT-532还可以增强F508del突变的CFTR，并专门针对突变CFTR而不是其他膜蛋白。‘ VRT-532 (CFpot-532)是CFTR 的有效调节剂，常用于CFTR 缺陷引起的囊性纤维化(CF)研究。降解囊性纤维化中积累的硫酸化糖胺聚糖(gag)所必需的Arylsulfatase B (ARSB)，小分子调节剂VRT-532对CFTR 的修饰能增加ARSB 并减少4-硫酸软骨素的积累。 ’ A mutated G551D CFTR corrector. VRT-532 mediates G551D-CFTR by enhancing ATP affinity of the mutant. VRT-532 also potentiates F508del mutated CFTR and works specifically on mutant CFTR rather than other membrane proteins.
