Article

AAV-mediated progranulin delivery to a mouse model of progranulin deficiency causes T cell-mediated hippocampal degeneration

2018-04-27

Abstract excerpt

Adeno-associated virus (AAV)-mediated gene replacement is emerging as a safe and effective means of correcting single-gene mutations, and use of AAV vectors for treatment of diseases of the CNS is increasing. AAV-mediated progranulin gene ( GRN ) delivery has been proposed as a treatment for GRN -deficient frontotemporal dementia (FTD) and neuronal ceroid lipofuscinosis (NCL), and two recent studies using focal...

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Literature Corpus work: 09b2345c-db9f-5ace-8c80-f61e3b3e5479
DOI: 10.1101/308692

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