Identification and Generation of Long Term Repopulating Human Muscle Stem Cells from Human Pluripotent Stem Cells

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Research Objective We will molecularly and functionally define muscle stem cells in human muscle in development, juvenile and adult and develop strategies to generate the most regenerative muscle stem cells…

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A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs

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Research Objective Develop a tool that facilitates rapid, cost effective development of optimized GMP-grade hPSC differentiation into functional DA neurons and apply this device to a cohort of PD patient-derived…

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CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease

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Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative…

Continue ReadingCRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease