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Therapeutic Target for Polycystic Kidney Disease Identified

September 19, 2022

Via: GEN

Researchers have reduced the formation and growth of kidney cysts in autosomal dominant polycystic kidney disease (ADPKD) models by blocking the binding of a microRNA called miR-17 to the PKD1 and PKD2 transcripts. The findings pave the way for a therapeutic strategy with the potential to arrest or cure ADPKD.

“For more than 25 years, we have known that ADPKD is caused by mutations of PKD1 or PKD2 genes. Yet, no therapeutic strategy exists to go after these root causes,” said Vishal Patel, MD, associate professor of internal medicine in the division of nephrology at UT Southwestern. Patel’s team led the work, which was published in Nature Communications in an article entitled “PKD1 and PKD2 mRNA cis-inhibition drives polycystic kidney disease progression.”

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