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…
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…
Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants
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…
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…
Mechanisms of Transcription Factor Haploinsufficiency in Human Congenital Heart Disease
Research Objective We aim to solve a 30 year old problem, which is to understand using human stem cells how certain genetic mutations cause human disease. Impact We currently don't…
Mapping and modeling endothelial cell fate decisions for pulmonary arterial hypertension
Research Objective We will build a foundational model and experimental platform to catalog all genes that promote and protect against PAH, with the potential to extend to many other developmental…
Drug Discovery for Dilated Cardiomyopathy using Patient-Derived Human iPSCs
Research Objective Greenstone Biosciences will use patient-derived stem cells to discover safe and effective drugs for cardiovascular disease. Impact The use of patient-derived stem cells to discover novel drug targets…
Modulating cardiac myosin heavy chain isoform expression for treating cardiomyopathies
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis
Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive…
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