Article

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

2024-04-19

Abstract excerpt

<h4>Background</h4> 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 cross the blood-brain barrier (...

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Literature Corpus work: d50f98a3-4d1f-5669-a5e0-4e0fd39c0f6c
DOI: 10.1101/2024.04.15.589486

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