Article
Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivo.
Neurobiology of disease - 1 Jul 2018
Schultz Mark L, Tecedor Luis, Lysenko Elena, Ramachandran Shyam, Stein Colleen S, Davidson Beverly L
Abstract excerpt
The neuronal ceroid lipofuscinoses are a class of inherited neurodegenerative diseases characterized by the accumulation of autofluorescent storage material. The most common neuronal ceroid lipofuscinosis has juvenile onset with rapid onset blindness and progressive degeneration of cognitive processes. The juvenile form is caused by mutations in the CLN3 gene, which encodes the protein CLN3. While mouse models of...
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