Identification of chemical and pharmacological chaperones for correction of trafficking- deficient mutant CNGA3 channels

We describe a novel luminescence-based assay to detect the surface expression of mutant, trafficking deficient CNGA3 channels based on the calcium sensitive photoprotein aequorin. Using this assay for a compound screening, we identified novel chemical and pharmacological chaperones restoring the surface localization of mutant trafficking-deficient CNGA3 channels. The results from our work may serve as starting point for the development of potent compounds rescuing trafficking deficiencies in the autosomal-recessively inherited retinal disease achromatopsia.PMID:33827965 | DOI:10.1124/molpharm.120.000180
Source: Molecular Medicine - Category: Molecular Biology Authors: Source Type: research