A potential drug treatment for autosomal dominant polycystic kidney disease (AD-PKD)– a genetic disorder that causes the kidneys to swell with multiple cysts and can eventually lead to organ failure – has shown promising results in animal testing. A study describing the drug’s development and testing was published on September 12, 2019 in Nature Communications (https://www.nature.com/articles/s41467-019-11918-y). (Note: Image shows polycystic kidney at left and normal kidney at right). The study shows an approximately 50 percent reduction in kidney size in afflicted mice following treatment. The drug is now in early clinical trials in human subjects, said Dr. Vishal Patel, Associate Professor of Internal Medicine at the University of Texas (UT) Southwestern Medical Center and senior author of the study. The open-access article is titled “Discovery and Preclinical Evaluation of Anti-miR-17 Oligonucleotide RGLS4326 for the Treatment of Polycystic Kidney Disease.” Autosomal dominant polycystic kidney disease (AD-PKD) affects approximately 12 million people worldwide, with half developing end-stage kidney disease by age 60, according to the study. “Once the kidneys have failed, the only options for survival are dialysis or a kidney transplant,” Dr. Patel said.
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