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…
Research Objective We will generate insights about mechanisms by which 𝛼-synuclein leads to neurodegeneration of the forebrain and substantia nigra, regions affected in Lewy body dementias and Parkinson disease. Impact…
Research Objective This project will identify mechanisms by which immune cells regulate human brain development, and use stem cell derived models to identify pathological changes induced by congenital virus infection.…
Research Objective Human and animal models of NuRD-deficiency will identify NuRD-subtype function in context of neurogenesis. Multi-omic studies will identify/quantify molecular and cellular changes in NuRD-deficiency. Impact NuRD-deficiency causes several…
Research Objective We will identify the risk factors underlying viral infections of the fetal brain using a novel human stem cell-based platform that has the potential to accelerate basic and…
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders.…
Research Objective This study will use pluripotent stem cells derived from patients to determine why Intellectual Disabilities caused by mutations in chromatin regulatory proteins leads to neuronal defects. Impact Our…
Research Objective We are building a "virtual molecular microscope" where anyone can quickly visualize a very recent, high-throughput molecular assay, single-cell RNA-seq and spatial gene expression studies Impact Currently, a…
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…
Research Objective We will generate an RNA activation map of human stem cell derived microglia activation states following brain injury to then test a new gene-edited microglia peptide delivery mechanism.…
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