Human Stem Cell Use: iPS Cell
Functional genomics to study cellular convergence across ASD risk genes in neurodevelopment
Research Objective Our objective is to enable scalable genetic screening to study how neurogenesis is impacted by risk genes implicated in human psychiatric disorders. Impact We will develop and apply state-of-the-art genomic analysis to seek mechanisms and disease modifying solutions. Major Proposed Activities Identifying and validating efficient gRNA for gene editing Identify risk gene effects […]
Establishment of a novel approach to systematically study the dynamic organization of protein complexes in stem cells
Research Objective We focus on hiPSCs pluripotency and neurodifferentiation to develop a novel framework to allow simultaneous identification of multiple interactions between proteins and between proteins and the genome Impact Our framework will allow high-throughput queries of the organization and functionality of proteins and shift the focus towards unprecedented, multi-dimensional studies of the cellular complexity. […]
Understanding Chemotherapy-Induced Peripheral Neuropathy Mechanisms using CRISPRi and Chemical Screens in Human iPSC-Derived Sensory Neurons
Research Objective The research objectives are to identify causal genes for chemotherapy-induced mitochondrial toxicity and neurodegeneration in sensory neurons and drugs that target this toxicity. Impact These studies will open the possibility for genetic or drug targeting to prevent and treat drug-induced peripheral neuropathies and possibly neuropathies caused by disease or inherited. Major Proposed Activities […]
The role of WNT and BMP signaling pathways in iPSC to iTenocyte step-wise differentiation for tendon repair
Research Objective Development-inspired differentiation will enable efficient and specific generation of tenocytes that can repair tendon injury, restore dysfunctional tissue, and prevent long term effects. Impact This study will eliminate heterogenous differentiation of pluripotent stem cells and will results in high yield and unified tenogenic phenotype. Major Proposed Activities Establish the mechanism by which Wnt […]
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 for cardiovascular disease, identify drug candidates, and establish clinical trial in a dish to evaluate drug safety and efficacy. Major Proposed Activities Use patient-derived induced […]
Developing a Human Model of Sporadic ALS Using Machine Learning and Robotic Microscopy
Research Objective We will develop the first human stem cell model of sporadic ALS (sALS) to identify disease mechanisms in the most common form of ALS and to discover drugs to treat the vast majority of ALS patients. Impact Failure of drugs to treat sALS may be due to the use of models of familial […]
Developing a microglia replacement therapy
Research Objective To develop a new cell therapy for the brain by transplanting the brain's immune cells Impact The brain cell therapy we envision could be used to treat rare genetic metabolic diseases, Multiple Sclerosis, brain tumors, as well as common neurodegenerative diseases including Alzheimer's disease. Major Proposed Activities Finding the best source of human […]
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 identify treatments for genetic diseases, but currently less than 1% of identified mutations have a known function. Major Proposed Activities Identify optimal conditions for SNV […]
Development of universal off-the-shelf iPSC derived dendritic cells for use as patient specific anti-tumor vaccine
Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the genetics of the patient and their cancer. Impact Success would provide proof-of-principle of a novel cell based therapy for cancers broadly. It is expected to […]
Control of OCT4 abundance and function in human stem cells
Research Objective Our work will reveal an essential pathway that establishes precise levels of the OCT4 transcription factor and protects stem cell identity despite genetic or environmental stress. Impact Our work will enhance our ability to generate and expand high-quality induced pluripotent stem cells from older patients or patients of neurodegenerative disease for use in […]