Novel Therapeutics:
Precision RNA Repair for Polycystic Kidney Disease
Vishal Patel, M.D.
- Professor of Internal Medicine, Division of Nephrology, UT Southwestern Medical Center
Dr. Patel is a nephrologist whose lab uncovered how a specific microRNA family (miR-17) drives cyst growth in polycystic kidney disease and showed that blocking this signal can rescue PKD1/PKD2 function in preclinical models. Building on that work and prior collaboration with Regulus Therapeutics, his Discovery Track focuses on new RNA drugs that precisely “repair” PKD1 signaling by targeting regulatory motifs in the PKD1 3’ UTR, using the patient’s remaining healthy gene copy. The goal is a kidney-targeted, programmable oligonucleotide therapy that slows or halts cyst growth with fewer systemic side effects than chronic small-molecule treatments. This concept is inherently modular and could extend to other dominant genetic kidney and systemic diseases where one working gene copy remains. If successful, the program positions UTSW as a leader in RNA-based gene repair for PKD and creates a platform that is attractive for both pharma partnering and a dedicated NewCo.
