Endothelial and neuronal engagement by AAV-BR1 alleviates neurological symptoms and cholesterol deposition in a mouse model of Niemann-Pick type C2
Charlotte Laurfelt Munch Rasmussen, Christian W. Heegaard, Maj Schneider Thomsen, Eva Hede, Bartosz Laczek, Jakob Körbelin, Louiza Bohn Thomsen, Markus Schwaninger, Torben Moos, Annette Burkhart
AAV-BR1-NPC2 gene therapy reduces cholesterol deposition and neurological symptoms in NP-C2 mice by delivering NPC2 to brain endothelial cells and neurons.
bioRxiv · Apr 15, 2024 · PreprintNeuroscience