AAV mediated delivery of a novel anti-BACE1 VHH reduces Abeta in an Alzheimer's disease mouse model

• Published in: bioRxiv
• Main Authors: Rincon, Melvin, Zhou, Lujia, Marneffe, Catherine, Voytyuk, Iryna, Wouters, Yessica, Dewilde, Maarten, Duqué, Sandra I, Vincke, Cécile, Levites, Yona, Golde, Todd E, Muyldermans, Serge, De Strooper, Bart, Holt, Matthew G
• Format: Paper
• Language: English
• Published: Cold Spring Harbor Cold Spring Harbor Laboratory Press 13.07.2019
• Subjects: Alzheimer's disease; Amyloidosis; Central nervous system; Expression vectors; Gene transfer; Nanobodies; Neurodegenerative diseases; Neurological diseases; Parenchyma; β-Site APP-cleaving enzyme 1

Single domain antibodies (VHH) are potentially disruptive therapeutics, with important biological value for the treatment of several diseases, including neurological disorders. However, VHH have not been widely used in the central nervous system (CNS), as it is hard to reach therapeutic levels, both...