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…
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.…
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…
Allele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes
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…
Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis
Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS…
Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies
Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a…
Development of universal off-the-shelf iPSC derived dendritic cells for use as patient specific anti-tumor vaccine
Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the…
- 1
- 2
- 3
- 4
- …
- 27
- Go to the next page
