Article
Targeting autophagy impairment improves the phenotype of a novel CLN8 zebrafish model.
Neurobiology of disease - 1 Jul 2024
Marchese Maria, Bernardi Sara, Ogi Asahi, Licitra Rosario, Silvi Giada, Mero Serena, Galatolo Daniele, Gammaldi Nicola, Doccini Stefano, Ratto Gian Michele, Rapposelli Simona, Neuhauss Stephan C F, Zang Jingjing, Rocchiccioli Silvia, Michelucci Elena, Ceccherini Elisa, Santorelli Filippo M
Abstract excerpt
CLN8 is an endoplasmic reticulum cargo receptor and a regulator of lysosome biogenesis whose loss of function leads to neuronal ceroid lipofuscinosis. CLN8 has been linked to autophagy and lipid metabolism, but much remains to be learned, and there are no therapies acting on the molecular signatures in this disorder. The present study aims to characterize the molecular pathways involved in CLN8 disease and, by...
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