Article

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

2024-12-19

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 cannot cross...

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Literature Corpus work: fa5d2bed-37f1-5e6a-a427-7b56b8cdeba2
DOI: 10.21203/rs.3.rs-5245293/v1

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