Disease Focus: Neurological Disorders
A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
CIRM ASCEND Center – Advancing Stem Cell Education and Novel Discoveries
The proposed CIRM ASCEND Center provides cutting-edge organoid-based services, offering consultation, optimized protocols, and genomic analysis. We also offer comprehensive training, workshops, and access to data, empowering researchers across California to excel in stem cell and organoid research. Our CIRM ASCEND Center benefits Californians by advancing regenerative medicine and healthcare. The Center’s services and training […]
A Center for Stem Cell Disease Modeling and Therapeutics
The goal of this project is to support the discovery and evaluation of novel therapeutics using stem cell-based models and drug and CRISPR screening. The facility will provide California researchers access to tools, technologies, and resources for regenerative medicine research. This project benefits California by helping accelerate research to find cures for major diseases of […]
A modular automation approach to stem cell modeling to increase throughput, reproducibility and access
This project enhances stem cell access, scalability, and collaboration. It offers characterized hPSC lines, CRISPR editing, and differentiation on automated platforms accelerating progress in biology, disease research, and regenerative medicine. The project benefits California by advancing regenerative medicine through diverse hPSC lines, potentially leading to novel treatments and addressing health disparities. It offers educational opportunities […]
Stem Cell-based Disease Modeling Shared Resource Laboratory
Our Shared Resource Laboratory will expand access to stem cell-derived models to support research and educational programs with two main goals: democratize access to state-of-the-art models and technology among a diverse scientific community and catalyze innovation in regenerative medicine. This Shared Resource Laboratory will expand access to stem cell-derived models to regions of Southern California […]
Lewy body dementia, 𝛼-synuclein, and cell-specific mechanisms of neurodegeneration
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 If the proposed studies are successfully achieved, they will impact our understanding of Lewy body dementias specifically and 𝛼-synucleinopathies and neurodegeneration more broadly. Major Proposed […]
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. Impact Neurodevelopmental disorders caused by immune cell perturbations will be impacted by the fundamental understanding of how immune system controls normal development of the brain. […]
Village-based identification of human risk factors for viral neuropathogenesis
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 translational discoveries. Impact Our work will identify the biological factors that influence inter-individual differences in susceptibility and immune response to neurotropic viruses, which could inform […]
Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment
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 neurodevelopmental disorders (NDDs), our work will identify and quantify cellular and molecular changes in human and mouse models of corticogenesis with NuRD deficiency. Major Proposed […]