Article

Outcomes of Progranulin Gene Therapy in the Retina are Dependent on Time of Delivery

2021-02-25

Abstract excerpt

Neuronal ceroid lipofuscinosis (NCL) is a family of neurodegenerative diseases caused by mutations to genes related to lysosomal function. One variant, CNL11, is caused by mutations to the gene encoding the protein progranulin. Primarily secreted by microglia, progranulin regulates neuronal lysosomal function once endocytosed. Absence of progranulin causes cerebellar atrophy, seizures, ataxia, dementia and vision...

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Literature Corpus work: 9251194b-474f-539c-ac27-309a7792f118
DOI: 10.1101/2021.02.24.432570

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