Stem cells and stem cell-derived neurons as a model for studying Alzheimer's Disease
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.
Alzheimer’s Disease-relevant neuronal model that is homogenous and highly physiological
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.
Lead Inventor:
Applications:
- Cell-based, high-throughput assays to screen compounds for the treatment of Alzheimer’s Disease.
- Research tool for identifying therapeutic targets and pathways involved in Alzheimer’s Disease.
Advantages:
- Establishes a highly relevant and renewable AD neuronal cell model
- Contains AD related cellular machinery and can respond appropriately to pharmacological manipulations
- Generates physiologically relevant synapses between AD neurons
Patent Information:
Patent Pending (WO/2012/122405
Patent Pending (US 20140171380)
Tech Ventures Reference: IR 2917
Related Publications:
- McIntire LB, Landman N, Kang MS, Finan GM, Hwang JC, Moore AZ, Park LS, Lin CS, Kim TW. “Phenotypic assays for β-amyloid in mouse embryonic stem cell-derived neurons.” Chem Biol. 2013 Jul 25;20(7):956-67.
- Kim TW. “Drug repositioning approaches for the discovery of new therapeutics for Alzheimer's disease.” Neurotherapeutics. 2015 Jan;12(1):132-42.