Site-specific gene editing in hematopoietic stem cells as an anti-HIV therapy
The overall goal of this proposal is to develop new methods and technologies to improve our ability to engineer hematopoietic stem cells. These are the adult stem cells found in…
Center of Excellence for Stem Cell Genomics – Stanford
The Center of Excellence in Stem Cell Genomics will bring together investigators from seven major California research institutions to bridge two fields – genomics and pluripotent stem cell research. The…
Site-specific integration of Lmx1a, FoxA2, & Otx2 to optimize dopaminergic differentiation
The objective of this study is to develop a new, optimized technology to obtain a homogenous population of midbrain dopaminergic (mDA) neurons in a culture dish through neuronal differentiation. Dopaminergic…
Directed Evolution of Novel AAV Variants for Enhanced Gene Targeting in Pluripotent Human Stem Cells and Investigation of Dopaminergic Neuron Differentiation
Human embryonic stem cells (hESCs) and induced pluripotent stem (iPS) cells have considerable potential as sources of differentiated cells for numerous biomedical applications. The ability to introduce targeted changes into…
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…
