Research Objective The proposed research will address key knowledge gaps in small activating RNAs (saRNAs) and could overcome bottlenecks limiting the broader adoption of this technology for regenerative medicine Impact…
Research Objective To integrate emulsion microfluidics-based encapsulation with AI-powered imaging to develop a high-throughput pipeline for stem cell-based embryo models. Impact We will overcome key bottlenecks to drive breakthroughs in…
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…
Research Objective We study how the inactive X chromosome aberrantly reactivates in female pluripotent stem cells and develop ways to prevent it, enabling accurate modeling of female biology for research…
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…
Research Objective Our project will identify molecular and cellular changes induced by specific genetic variants implicated in schizophrenia and autism spectrum disorder in stem cells, neuroprogenitor cells and neurons. Impact…
Research Objective We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.…
Research Objective This proposal leverages pluripotent stem cell models to understand the principles by which changes in gene and protein dosage affect human neural and facial progenitor development and cause…
Research Objective We seek to develop a more accessible method of genome editing of blood stem cells directly in the body, so that the treatment can be safer and less…
Research Objective We aim to solve a 30 year old problem, which is to understand using human stem cells how certain genetic mutations cause human disease. Impact We currently don't…
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