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PC1-CTT is a recombinant polypeptide corresponding to the final 200 amino acid residues of the C-terminal tail of polycystin-1 (PC1). It is being investigated as a potential gene or peptide therapy for the treatment of autosomal dominant polycystic kidney disease (ADPKD). PC1-CTT translocates to the mitochondria and nucleus, where it interacts with the mitochondrial enzyme nicotinamide nucleotide transhydrogenase (NNT) to reduce oxidative stress, alter redox modulation, increase mitochondrial mass, and suppress the cystic phenotype. It also interacts with beta-catenin in the nucleus to inhibit canonical Wnt signaling, thereby suppressing cystogenesis.
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