Development of in vitro and in vivo functional human synthetic kidney organoid (hSKO) model as a platform technology for kidney research

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Research Objective Development of a human stem cell-derived, spatially-patterned, mature and functional human synthetic kidney organoid (hSKO) model as a platform technology for basic and translational kidney research. Impact Lack…

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Providing a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy

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Research Objective AAV-SPL 2.0 is a gene therapy cure for SPLIS, a lethal childhood disorder of metabolism that causes kidney failure. Our gene therapy may also work in more common…

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Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies

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Research Objective Approximately 20,000 babies are born annually with kidney disease; the long-term outcome is poor. These studies address new ways to develop mini-kidney structures for transplantation to induce repair.…

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