Article

Carboxy-terminal blockade of sortilin binding enhances progranulin gene therapy, a potential treatment for frontotemporal dementia

2024-09-16

Abstract excerpt

Frontotemporal dementia is commonly caused by loss-of-function mutations in the progranulin gene. Potential therapies for this disorder have entered clinical trials, including progranulin gene therapy and drugs that reduce progranulin interactions with sortilin. Both approaches ameliorate functional and pathological abnormalities in mouse models of progranulin insufficiency. Here we investigated whether modifying...

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Literature Corpus work: 1856d967-9535-5829-99cb-df82afe78ec0
DOI: 10.1101/2024.09.15.613118

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Carboxy-terminal blockade of sortilin binding enhances progranulin gene therapy, a potential treatment for frontotemporal dementiaDOI 10.1101/2024.09.15.613118Read

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