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A research group of Professor Kenji Osafune has identified a drug candidate for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and published a research paper about that achievement

A research group of our Director and Chief Scientific Advisor, Professor Kenji Osafune, succeeded in creating a disease model that reproduces the characteristics of autosomal dominant polycystic kidney disease (ADPKD)(1) using organoids(2) created from iPS cells, and discovered a potential therapeutic drug for ADPKD.

The research results were published in “Cell Reports” on December 1, 2023.

Title:“Human iPSC-derived renal collecting duct organoids model cystogenesis in ADPKD”

Press Release from Center for iPS Cell Research and Application, Kyoto University
https://www.cira.kyoto-u.ac.jp/e/pressrelease/news/231201-010000.html

Note (1) Autosomal Dominant Polycystic Kidney Disease (ADPKD)
ADPKD is an incurable hereditary disease in which numerous cysts gradually develop and enlarge in both kidneys, and kidney dysfunction progresses, leading to end-stage renal failure requiring artificial dialysis or kidney transplantation. In addition to the kidneys, cysts can also occur in the liver and pancreas, and it is known that cysts are frequently associated with systemic blood vessel abnormalities, high blood pressure, cerebral aneurysms, and heart valve abnormalities. Although the drug tolvaptan has been approved, there is no cure for ADPKD.

Note (2) Organoid
A small organ created three-dimensionally in vitro.