High-throughput screening to identify modulators of PKD2 function

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The NCATS Early Translation Branch (ETB) hosts a broad and comprehensive program for the discovery of drug candidates directed towards rare diseases and pharmacological tools to probe the function of the human genome. ETB conducts research to understand the underlying principles driving the translation of basic research discoveries into tangible improvements in human health. Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in transmembrane proteins PKD1 (PC1) or PKD2 (PC2). PC1 and PC2 subunits form a cation channel at the plasma membrane, and function can be disrupted by disease-causing mutations. During this period, the collaborative team developed and optimized a high-throughput Fluo8 calcium assay to identify regulators of PC1/2, and orthogonal assay development is ongoing.