CIRM Center for Neuropsychiatric Stem Cell Proteomics
Research Objective This project will interrogate interactions, distribution, and function of high-confidence neuropsychiatric disorder risk proteins, and identify convergent pathobiology of patient genetic variants. Impact Datasets and stem cell resources…
Hearing the Silence: Genome-wide Mapping of Cell-Type-Specific Silencers in the Developing Human Brain
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…
A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency
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.…
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…
Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis
Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the…
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…
Engineering AAV capsids for transduction of neural and muscle stem cells
Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and…
A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering
Research Objective We propose to discover genome- and epigenome-edited allogeneic T cells engineered to selectively target JCV as a potentially lifesaving treatment for progressive multifocal leukoencephalopathy (PML). Impact If successful,…