In neurons and beyond: how protein interactions shape the cellular response to Huntington’s Disease

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Research Objective New knowledge relating to protein-protein interactions that drive vulnerability of different cell types to neurodegeneration disease mutations using Huntington's disease as a paradigm. Impact The studies will inform…

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Unraveling nuclear Tau functions using age-equivalent human induced neurons from healthy aging donors and tauopathy patients

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Research Objective This project investigates how normal tau protein protects aging neurons. We'll use aging human neurons to understand how tau helps repairing DNA and why this protection fails disease.…

Continue ReadingUnraveling nuclear Tau functions using age-equivalent human induced neurons from healthy aging donors and tauopathy patients

Allele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes

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Research Objective We are building a foundational platform for developing genome editing technologies that increase patient coverage by 20-40X and can be applied to over 700 genetic diseases in diverse…

Continue ReadingAllele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes

Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms.

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Research Objective Study the neurological disorder Rett syndrome using neurons derived from genetically engineered human pluripotent stem cells and develop platforms to screen drugs for this disease in live cells.…

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