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A novel strategy for delivering Niemann‐Pick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus

Treating central nervous system (CNS) diseases is complicated by the incapability of numerous therapeutics to cross the blood–brain barrier (BBB), mainly composed of brain endothelial cells (BECs). Genetically modifying BECs into protein factories that supply the CNS with recombinant proteins is a p...

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Detalles Bibliográficos
Autores principales: Rasmussen, Charlotte Laurfelt Munch, Hede, Eva, Routhe, Lisa Juul, Körbelin, Jakob, Helgudottir, Steinunn Sara, Thomsen, Louiza Bohn, Schwaninger, Markus, Burkhart, Annette, Moos, Torben
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10084444/
https://www.ncbi.nlm.nih.gov/pubmed/35554935
http://dx.doi.org/10.1111/jnc.15621