Biochem/physiol Actions

QX77 is a chaperone-mediated autophagy (CMA) activator derived from the atypical retinoid AR7 that activates CMA by antagonizing retinoic acid receptor-α (RARα) signaling. QX77 rescues defective trafficking & lysosomal localization of the CMA receptor LAMP2A in cystinotic Ctns^-/- MEFs and CTNS-KO human proximal tubule cells (PTCs) by restoring Rab11 expression and Rab11-positive vesicles trafficking to the level seen in wild-type cells (20 µM, 48 hrs). CMA activation by QX77 treatment (20 µM, 5 days) also significantly reduces toxic α-synuclein (α-syn) accumulation in PD patients iPSCs-derived astrocytes with LRRK2 G2019S mutation.