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- W4313250952 abstract "Front cover Brain endothelial cells (BECs) denote the blood-brain barrier and limit the passage of therapeutic proteins from the circulation to the brain thereby challenging the treatment of neurological diseases. Genetically modifying BECs into protein factories that supply the CNS with recombinant proteins is a promising approach to overcome this hindrance, especially in genetic diseases, like Niemann Pick disease type C2 (NPC2). Here we prove evidence of viral transduction of primary BECs leading to secretion of fully functional NPC2 protein resulting in therapeutic efficacy with a reduction in cholesterol accumulation in NPC2 deficient fibroblasts. Furthermore, intravenous injection of the brain-specific AAV-BR1 viral vector in healthy BALB/c mice leads to a widespread genetic modification of brain capillaries with the synthesis of recombinant eGFP and NPC2 proteins. eGFP and NPC2 were also occasionally expressed in neurons suggesting transport of the vector across the BBB. The gene therapy strategy using the AAV-BR1 virus denotes a promising strategy for future treatment of NPC2. Image content Representative image of transduced neurons in the hippocampal region of mice eight weeks post-injection of the brain-specific AAV-BR1 virus encoding the eGFP and NPC2 proteins. The image shows co-localization of eGFP (green) and the neuronal cell marker (NeuN) (red). Nuclei are counterstained with DAPI (blue). Scale bar 50 μm. Read the full article ‘A novel strategy for delivering Niemann-Pick type C2 proteins across the blood–brain barrier using the brain endothelial-specific AAV-BR1 virus’ by C. L. M. Rasmussen, E. Hede, L. J. Routhe, J. Körbelin, S. S. Helgudottir, L. B. Thomsen, M. Schwaninger, A. Burkhart, T. Moos. 2023, vol.164 (1), pp. 6–28) on doi:10.1111/jnc.15621" @default.
- W4313250952 created "2023-01-06" @default.
- W4313250952 date "2023-01-01" @default.
- W4313250952 modified "2023-09-25" @default.
- W4313250952 doi "https://doi.org/10.1111/jnc.v164.1" @default.
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