BMS-708163 and Nilotinib restore synaptic dysfunction in human embryonic stem cell-derived Alzheimer's disease models

摘要: Alzheimer’s disease (AD) is the most common form of dementia. Cellular AD models derived from human pluripotent stem cells are promising tools in research. We recently developed embryonic cell-derived which overexpress mutant Presenilin1 genes, and exhibit phenotypes, including synaptic dysfunction. In this study, we found that our showed reduced levels RAB3A SV2B proteins pre-synapses, a possible cause electrophysiological abnormalities. Through screening chemical compounds using models, have identified Aβ peptide inhibitors decrease concentration culture supernatant. Among these, BMS-708163 Nilotinib were to improve expression recover function models. These results suggest materials for discovery drugs target pre-synaptic function.