UT Southwestern Researchers Identify Gene Therapy Target for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Blocking the inhibition of PKD1 and PKD2 gene expression by deleting a binding site for microRNAs hindered the formation and growth of kidney cysts in autosomal dominant polycystic kidney disease (ADPKD) models, UT Southwestern (UTSW) researchers reported. The findings, published on September 15, 2022 in Nature Communications, suggest a strategy for gene therapy with the potential to arrest or cure ADPKD. The open-access article is titled “PKD1 and PKD2 mRNA Cis-Inhibition Drives Polycystic Kidney Disease Progression.”