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Early and Long

Enzyme ANKMY2 action on cilia lengthening underlies formation of cysts in polycystic kidney disease – potential target for treatment

19 March 2026

Early and Long

Intervene at the early stages of a disease and you might never have to face the later consequences. A study investigated what triggers early cyst formation in polycystic kidney disease, which occurs when the gene Pkd1 stops working (black cyst regions pictured in the mouse kidney, top). Researchers found that a protein called ANKMY2 directs signalling enzymes into a tiny cell structure called the primary cilium, where they produce a chemical messenger, cAMP, known to be involved in cyst growth. When ANKMY2 was removed, early cyst growth was reduced (bottom) and survival improved. Existing polycystic kidney disease treatments lower cAMP throughout the body, but this study suggests targeting ciliary signalling specifically may offer a more precise strategy. The researchers also found that cilia lengthen before visible cysts form, so could be a biomarker of early disease activity.

Written by Anthony Lewis

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