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.…
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…
RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis
Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive…
Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation
Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide…
Novel antisense therapy to treat genetic forms of neurodevelopmental disease.
Research Objective We propose to discovery and evaluate antisense gene therapy for specific mutations underlying debilitating or life-threatening neurodevelopmental diseases including epilepsy and autism syndromes. Impact The conditions are four…