Pkd1 mRNA stabilizing oligonucleotide

Pkd1 mRNA stabilizing oligonucleotide (also known as Pkd1-oligonucleotide or P1/P2) is a preclinical steric-blocking antisense oligonucleotide (ASO) developed by researchers at the University of Texas Southwestern Medical Center for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). The oligonucleotide is designed to target a specific six-nucleotide microRNA-17 (miR-17) binding motif within the 3' untranslated region (3'UTR) of the PKD1 mRNA. By binding to this motif, the oligonucleotide sterically hinders the interaction of miR-17 with the PKD1 transcript, thereby preventing miR-17-mediated translational repression and mRNA degradation. This action prolongs the half-life of PKD1 mRNA and enhances its translation, leading to increased levels of endogenous polycystin-1 (PC1) protein. In preclinical models of ADPKD, treatment with this oligonucleotide has been shown to reduce cyst size, improve mitochondrial metabolism, and mitigate pathogenic signaling pathways.

Other names

PKD1-targeting antisense oligonucleotidePKD-1-targeting antisense oligonucleotidePKD 1-targeting antisense oligonucleotidePKD1-targeting steric-blocking oligonucleotidePKD-1-targeting steric-blocking oligonucleotidePKD 1-targeting steric-blocking oligonucleotidePkd1 oligoPkd-1 oligoPkd 1 oligoPkd1-oligonucleotidePkd-1-oligonucleotidePkd 1-oligonucleotide