Modeling the genetic basis of psychopathology in schizophrenia and autism
Research Objective We propose to apply scalable platforms for the characterization of CNVs and genes coupled with detailed characterizations of neurodevelopment of the 4 major CNV loci in model systems…
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…
Global profiling of miRNA-based gene activation to enable a new category of genetic medicine
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…
High-Throughput Discovery of Embryo Formation Factors Using Stem Cell-Based Human Embryo Models
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…
Interrogation of tandem repeat variants contributing to neurodevelopmental and psychiatric traits using stem cell models
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…
Hearing the Silence: Genome-wide Mapping of Cell-Type-Specific Silencers in the Developing Human Brain
Research Objective We will develop genome editing tools to identify silencers that regulate neural stem cell fate, uncovering key DNA elements that guide neurodevelopment and are disrupted in neurodevelopmental diseases…
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…
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…
Neuroimmune interactions in the developing human brain
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.…