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

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