Laboratory for Cell and Gene Medicine: A partner in the California Cell and Gene Therapy Manufacturing Network

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The Stanford LCGM supports process development and manufacturing for investigator initiated and select industry partner clinical trials. The objective of our proposal is to complete our project plan and enhance…

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A comprehensive biomanufacturing center solving bottlenecks in cell and gene therapy manufacturing to accelerate new therapies for California patients

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We aim to considerably expand access for all Californians to outstanding cell and gene therapy treatments for multiple patients. With operational enhancements to develop diverse talent from underserved communities, our…

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Next generation stem cell transplantation approaches for pediatric neurodegenerative disorders

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Research Objective A complete methodology for high efficiency, minimal toxicity hematopoietic stem cell transplantation for treatment of pediatric neurodegenerative disorders. Impact Improve treatment and outcomes for patients with neurodegenerative disorders…

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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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Hearing the Silence: Genome-wide Mapping of Cell-Type-Specific Silencers in the Developing Human Brain

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Research Objective We will develop genome editing tools to identify silencers that regulate neural stem cell fate, uncovering key DNA elements that guide neurodevelopment and are disrupted in neurodevelopmental diseases…

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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…

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