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…
Use of stem cells in human disease modeling has reached a broader scientific audience of underserved, marginalized communities. Researchers/scholars have comprehensive training in iPSC generation, genetic modification, and differentiation techniques…
Research Objective The research will generate the know-how for replacing lost or injured bile ducts in the liver by reprogramming an abundant and related cell type that normally has a…
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…
Research Objective Develop a high-throughput platform combining iPSC-derived cardiac organoids and CRISPR base editing to functionally assess missense variants in hypertrophic cardiomyopathy. Impact (i) Limited tools for functional interpretation of…
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…
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…
Research Objective We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.…
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…
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…
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