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…·Claire Russell
This study identifies pregnenolone as a therapeutic candidate for CLN2 disease by suppressing seizures and normalizing lysosomal function in tpp1-/- zebrafish.