One major obstacle in the discovery of drugs for Alzheimer’s Disease (AD) is the lack of a renewable, physiologically relevant neuronal model that accurately recapitulates the pathophysiology of the disease. This technology establishes a homogenous and renewable primary neuronal model for studying AD in vitro. This model could be used for high throughput screening of small molecules for drug discovery and testing, as well as a cell-based research platform to study and identify new cellular pathways and targets for the treatment of AD.
This technology describes a primary neuronal model developed from mouse embryonic stem cells. Specifically, embryonic stem cells are isolated from a mouse model of AD and are differentiated into neuronal cells to generate a physiologically relevant and renewable AD cell model. These cells contain the faulty cellular machinery observed in the neurons of AD patients, effectively recapitulating the pathophysiology of the disease. This model has the potential to be used for high throughput cell-based small molecule screens, to improve AD research and therapeutic testing.
The accuracy of the cell line in portraying AD was verified by morphological and functional assays. Results show that these cells behaved similarly to native diseased cells. Additionally, an assay using these cells has been developed and used to identify small molecule candidates capable of reducing amyloid β levels in the mouse-derived neuronal cells.
Patent Pending (WO/2012/122405
Patent Pending (US 20140171380)
Tech Ventures Reference: IR 2917