Translational epigenomics: dissecting cell type-specific function of neuropsychiatric risk genes in vivo
Research Objective Our objective is to enable scalable genetic screening to study how different cell types and epigenetic networks are impacted by risk genes implicated in human psychiatric disorders. Impact…
Identifying and Overcoming Roadblocks to Hearing Restoration Using Human Induced Pluripotent Stem Cells
Research Objective To utilize human iPSCs as an in vitro model of hearing loss and overcome obstacles to hair cell regeneration by prompting post-mitotic supporting cells to re-enter the cell…
Unraveling nuclear Tau functions using age-equivalent human induced neurons from healthy aging donors and tauopathy patients
Research Objective This project investigates how normal tau protein protects aging neurons. We'll use aging human neurons to understand how tau helps repairing DNA and why this protection fails disease.…
A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency
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.…
Mechanisms underlying dosage sensitivity in developmental disorders
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…
Harnessing developmental biology to achieve safe and efficient in vivo genome editing of HSCs
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…
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