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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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Principais autores: 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
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2025-01-01
Serier:Fluids and Barriers of the CNS
Fag:
AAV-BR1
Blood–brain barrier
Viral gene therapy
Niemann-Pick type C2 disease
NPC2
Cross-correction
Online adgang:https://doi.org/10.1186/s12987-025-00621-4
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