Therapeutic/Technology: Model system development
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 […]
Shared Resources Laboratories to Enhance In Vitro Stem Cell Modeling and Training
Our broad objective is to expand access to equipment, services, and training within our areas of excellence. Through knowledge sharing and collaboration, we aim to add value to the CIRM SRL network. We seek to build on our historical success and foster excellence in California stem cell research. The SRL will provide access to specialized […]
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 […]
Modeling and understanding alveolar hypoplasia in Down syndrome using iPSCs-derived alveolar type II cells
Research Objective Understanding alveolar progenitor cell defects in T21 and the genes/pathways associated with them will allow for developing therapeutic approaches for individuals with DS. Impact Although trisomy 21 affects multiple organ system, respiratory complications are the major cause of death in kids and adults with DS. The causes of lung disease in DS remain […]
Characterization and applications of human blastoids for understanding early human embryogenesis
Research Objective Our work will yield an improved stem-cell based embryo model that we will explore with various omics approaches and genetic screens to gain insights into the pathways that control human embryos. Impact An improved stem cell-based embryo model is a crucial step for in-depth studies of human development and will enhance our ability […]
An interactive data resource for hypothesis testing in stem cell single-cell gene expression and validation of the results with brain organoids
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 lot of data has been published, hundreds of datasets on the cerebral cortex alone, but it takes hours to convert the datasets and look at […]
Developing a Human Model of Sporadic ALS Using Machine Learning and Robotic Microscopy
Research Objective We will develop the first human stem cell model of sporadic ALS (sALS) to identify disease mechanisms in the most common form of ALS and to discover drugs to treat the vast majority of ALS patients. Impact Failure of drugs to treat sALS may be due to the use of models of familial […]
Development of a stem-cell based approach to interpret global effects of genetic variants contributing to neurodevelopmental disease risk
Research Objective We are developing a strategy to characterize the disease-relevance of hundreds of mutations across diverse genetic backgrounds using stem cells Impact Understanding how mutations impact cellular function can identify treatments for genetic diseases, but currently less than 1% of identified mutations have a known function. Major Proposed Activities Identify optimal conditions for SNV […]