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

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 t...

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Detaylı Bibliyografya
Asıl Yazarlar:	Charlotte Laurfelt Munch Rasmussen, Signe Frost Frederiksen, Christian Würtz Heegaard, Maj Schneider Thomsen, Eva Hede, Bartosz Laczek, Jakob Körbelin, Daniel Wüstner, Louiza Bohn Thomsen, Markus Schwaninger, Ole N. Jensen, Torben Moos, Annette Burkhart
Materyal Türü:	Artigo
Dil:	Inglês
Baskı/Yayın Bilgisi:	BMC 2025-01-01
Seri Bilgileri:	Fluids and Barriers of the CNS
Konular:	AAV-BR1 Blood–brain barrier Viral gene therapy Niemann-Pick type C2 disease NPC2 Cross-correction
Online Erişim:	https://doi.org/10.1186/s12987-025-00621-4
Etiketler:	Etiketle Etiket eklenmemiş, İlk siz ekleyin!

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

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