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Bartosz Laczek

As seen in: BioRxiv
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Articles

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

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By Christian Würtz Heegaard, Eva Hede, Schneider Thomsen, Bartosz Laczek
| BioRxiv
Abstract Background: 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 (BBB).
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