Monoallelic Loss of Human PKD1 Causes ADPKD in a Fully Humanized Mouse Model (KIDNEY WEEK 2026) - Oct 2, 2026 - Abstract #TH-OR055; Pres time: Oct 22, 2026; 04:30 PM - 04:40 PM; Location: Mile High Ballroom 4D, Convention Center; "To test this axis pharmacologically, Pkd1 H/- primary kidney cells and inducible haploinsufficient Ksp-rtTA;TetO-Cre; Pkd1 H/F (i- Pkd1 H/- ) mice were treated with the anti-miR-17 oligonucleotide RGLS4326...Rescue by 3'UTR deletion establishes 3'UTR-mediated regulation as causally required for pathogenesis, and attenuation by anti-miR-17 validates it as a tractable therapeutic target. The model enables studies of human allele-specific biology and direct preclinical testing of human-sequence therapeutics, including ASOs, siRNAs, and gene editors."
Preclinical • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • MIR17 • PKD1 • PRKD1
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Chunzi Song; Jesus A. Alvarez; Patricia Cobo-Stark; Mauricio Ostrosky Frid; Ronak Lakhia; Vishal Patel
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