In Utero Model to Assess the Fate of Transplanted Human Cells for Translational Research and Pediatric Therapies

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nfants with inherited blood diseases (such as sickle cell anemia, thalassemia, bleeding disorders) or other inherited metabolic disorders can be identified early in development using sophisticated diagnostic tests. Currently, the…

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Using patient-specific iPSC derived dopaminergic neurons to overcome a major bottleneck in Parkinson’s disease research and drug discovery

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The goals of this study are to develop patient-specific induced pluripotent cell lines (iPSCs) from patients with Parkinson’s disease (PD) with defined mutations and sporadic forms of the disease. Recent…

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Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments

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Research Objective We will uncover pathways through which ASD mutations cause disease and close the gap from disease research to therapeutic testing using organoids, primary human neurons, machine learning and…

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