Sentences with phrase «develops isogenic»
His lab develops isogenic human pluripotent stem cells and transgenic animals to model disease, with the goal of delineating novel approaches to influence outcomes for Huntington disease (HD) and Fragile X Syndrome (FXS), the most common genetic causes of dementia and intellectual disability, respectively.
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These isogenic cell lines can be used not only to understand the cellular consequences of disease mutations, but also to perform high throughput genetic and pharmacological screens to both understand the underlying pathological mechanisms and to develop novel therapeutic agents to prevent or treat such diseases.