Therapeutic/Technology: Model system development
Mouse Models for Stem Cell Therapeutic Development
Stem cells have tremendous potential for treating human diseases, as they have the unique capacity to develop into any cell type in the body and to proliferate indefinitely. The development of new therapies based on the transplantation of human stem cells (HuSC) into patients is a major focus of California researchers. A critical step prior […]
Developing induced pluripotent stem cells into human therapeutics and disease models
Human embryonic stem cells (hESCs) can undergo unlimited self-renewal and differentiate into all the cell types in the human body, and thus hold great promise for cell replacement therapy. However, one major problem for hESC-based therapy is that the cells derived from hESCs will be rejected by the recipient and can only be tolerated under […]
Using patient-specific iPSC derived dopaminergic neurons to overcome a major bottleneck in Parkinson’s disease research and drug discovery
The goals of this study are to develop patient-specific induced pluripotent cell lines (iPSCs) from patients with Parkinson’s disease (PD) with defined mutations and sporadic forms of the disease. Recent groundbreaking discoveries allow us now to use adult human skin cells, transduce them with specific genes, and generate cells that exhibit characteristics of embryonic stem […]
Generation of disease models for neurodegenerative disorders in hESCs by gene targeting
The ability to target a specific locus in the mouse genome and to alter it in a specific fashion has fundamentally changed experimental design and made mice the preeminent model for studying human diseases . However, pathogenesis in humans have unique pathways that may not be revealed by only using mouse or other animal models. […]
Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments
Research Objective We will uncover pathways through which ASD mutations cause disease and close the gap from disease research to therapeutic testing using organoids, primary human neurons, machine learning and AAVs. Impact Our studies are impactful because outcomes will lead to therapeutic avenues to pursue for ASD treatment. 1 in 22 children in California is […]
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 […]
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 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 […]
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 […]
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 […]