Autologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cells

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Research Objective We are developing a durable patient-specific dopamine neuron replacement therapy for Parkinson's disease Impact Parkinson's disease is diagnosed when half of the dopamine (DA) neurons are lost from…

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Chemically engineered photoreceptors for vision restoration in retinal degeneration associated blindness.

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Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…

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A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering

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Research Objective We propose to discover genome- and epigenome-edited allogeneic T cells engineered to selectively target JCV as a potentially lifesaving treatment for progressive multifocal leukoencephalopathy (PML). Impact If successful,…

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Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients

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Research Objective We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1…

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