Whole-organism screening in a zebrafish model of CLN2 disease identifies pregnenolone as a modulator of lysosomal functions with anti-epileptic properties
Lisa Kiani, Gabriele Civiletto, Giulia Lizzo, Anselm A. Zdebik, Gini Brickell, Fahad Mahmood, Dionysios D Nalkos, Lucas Michaelides, Philip Eldridge, Emily Young, Hazel McPherson, Michelangelo Campanella, Philipp Gut, Claire Russell
This study identifies pregnenolone as a therapeutic candidate for CLN2 disease by suppressing seizures and normalizing lysosomal function in tpp1-/- zebrafish.
bioRxiv · Aug 26, 2025 · PreprintPharmacology & Toxicology