[HTML][HTML] ESCRT-mediated uptake and degradation of brain-targeted α-synuclein single chain antibody attenuates neuronal degeneration in vivo

B Spencer, S Emadi, P Desplats, S Eleuteri, S Michael… - Molecular Therapy, 2014 - cell.com
B Spencer, S Emadi, P Desplats, S Eleuteri, S Michael, K Kosberg, J Shen, E Rockenstein…
Molecular Therapy, 2014cell.com
Parkinson's disease and dementia with Lewy bodies are neurodegenerative disorders
characterized by accumulation of α-synuclein (α-syn). Recently, single-chain fragment
variables (scFVs) have been developed against individual conformational species of α-syn.
Unlike more traditional monoclonal antibodies, these scFVs will not activate or be
endocytosed by Fc receptors. For this study, we investigated an scFV directed against
oligomeric α-syn fused to the LDL receptor-binding domain from apolipoprotein B (apoB) …
Parkinson's disease and dementia with Lewy bodies are neurodegenerative disorders characterized by accumulation of α-synuclein (α-syn). Recently, single-chain fragment variables (scFVs) have been developed against individual conformational species of α-syn. Unlike more traditional monoclonal antibodies, these scFVs will not activate or be endocytosed by Fc receptors. For this study, we investigated an scFV directed against oligomeric α-syn fused to the LDL receptor-binding domain from apolipoprotein B (apoB). The modified scFV showed enhanced brain penetration and was imported into neuronal cells through the endosomal sorting complex required for transport (ESCRT) pathway, leading to lysosomal degradation of α-syn aggregates. Further analysis showed that the scFV was effective at ameliorating neurodegenerative pathology and behavioral deficits observed in the mouse model of dementia with Lewy bodies/Parkinson's disease. Thus, the apoB modification had the effect of both increasing accumulation of the scFV in the brain and directing scFV/α-syn complexes for degradation through the ESCRT pathway, leading to improved therapeutic potential of immunotherapy.
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