Endothelial and neuronal engagement by AAV-BR1 gene therapy alleviates neurological symptoms and lipid deposition in a mouse model of Niemann-Pick type C2
AAV-BR1 基因治疗促进内皮细胞和神经元的结合,可缓解尼曼匹克 C2 型小鼠模型中的神经症状和脂质沉积
期刊:Fluids and Barriers of the Cns
影响因子:5.9
doi:10.1186/s12987-025-00621-4
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 #