Researchers identify a gene therapy target for polycystic kidney disease

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 researchers reported. The findings, published in Nature Communications, suggest a strategy for gene therapy with the potential to arrest or cure ADPKD.