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Screening Assays Using Stem Cells and Stem Cell-Derived Neurons from Mouse Models of Alzheimer's Disease

机译:使用阿尔茨海默氏病小鼠模型中的干细胞和干细胞衍生的神经元进行筛选试验

摘要

Successful CNS drug discovery requires a scalable, highly physiological neuronal model. Using directed differentiation of mouse embryonic stem (mES) cells, including mES cells isolated from a mouse model of Alzheimer's disease (AD), a highly homogeneous primary neuronal model amenable to phenotypic assays for production and synaptotoxicity of amyloid β-peptide was developed. This model furnishes a highly physiological and AD-relevant platform suitable for high throughput small molecule and functional genetic screens, providing specific small molecule compounds identified by such screens.
机译:成功的CNS药物发现需要可扩展的高度生理神经元模型。利用小鼠胚胎干(mES)细胞(包括从阿尔茨海默氏病(AD)小鼠模型中分离的mES细胞)的定向分化,开发了一种高度均匀的初级神经元模型,该模型适合于表型测定法用于淀粉样β肽的产生和突触毒性。该模型提供了高度生理和与AD相关的平台,适用于高通量小分子和功能性遗传筛选,提供了通过此类筛选鉴定的特定小分子化合物。

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