Article
Progranulin as a therapeutic target in neurodegenerative diseases.
Trends in pharmacological sciences - 1 Aug 2022
Rhinn Herve, Tatton Nadine, McCaughey Stella, Kurnellas Michael, Rosenthal Arnon
Abstract excerpt
Progranulin (PGRN, encoded by the GRN gene) plays a key role in the development, survival, function, and maintenance of neurons and microglia in the mammalian brain. It regulates lysosomal biogenesis, inflammation, repair, stress response, and aging. GRN loss-of-function mutations cause neuronal ceroid lipofuscinosis or frontotemporal dementia-GRN (FTD-GRN) in a gene dosage-dependent manner. Mutations that reduce...
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