Article

Endothelial and neuronal engagement by AAV-BR1 alleviates cholesterol deposition in a mouse model of Niemann-Pick type C2

2024-04-19

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold> Patients with the genetic disorder Niemann-Pick type C2 disease (NP-C2) suffer from lysosomal accumulation of cholesterol causing both systemic and severe neurological symptoms. In a murine NP-C2 model, otherwise successful intravenous Niemann-Pick C2 protein (NPC2) replacement therapy fails to alleviate progressive neurodegeneration as infused NPC2 is unable to...

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Literature Corpus work: 32a57a74-ee76-5441-8be2-75ebd25112d6
DOI: 10.21203/rs.3.rs-4238772/v1

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