Therapeutic/Technology: Model system development

An interactive data resource for hypothesis testing in stem cell single-cell gene expression and validation of the results with brain organoids

Research Objective We are building a "virtual molecular microscope" where anyone can quickly visualize a very recent, high-throughput molecular assay, single-cell RNA-seq and spatial gene expression studies Impact Currently, a lot of data has been published, hundreds of datasets on the cerebral cortex alone, but it takes hours to convert the datasets and look at […]

Characterization and applications of human blastoids for understanding early human embryogenesis

Research Objective Our work will yield an improved stem-cell based embryo model that we will explore with various omics approaches and genetic screens to gain insights into the pathways that control human embryos. Impact An improved stem cell-based embryo model is a crucial step for in-depth studies of human development and will enhance our ability […]

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 […]

Towards a trophectoderm stem-cell model representing human blastocysts of the highest implantation potential

Research Objective To define a new reference for embryos and stem cell lines of the highest developmental potential and work towards a trophoblast stem cell model to study factors important for successful implantation Impact The molecular determinants of successful human blastocyst implantation remain unknown and trophectoderm stem cell models to study these embryo factors and […]

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 […]

Stem cell-based rapid identification of SARS-CoV-2 T cell epitopes and T cell receptors for therapeutic use

Research Objective We will identify SAR-CoV-2 T cell epitopes for vaccine development and specific TCRs for adoptive T cell therapy using a stem cell-based platform to generate specialized dendritic cells in vitro. Impact New methods to rapidly identify T cell epitopes would greatly accelerate development of vaccines and TCR-based therapeutics, and in the setting of […]

Safety “Clinical Trial” of the Cardiac Liability of COVID19 Polytherapy

Drug Discovery for Duchenne Muscular Dystrophy Using Patient-Derived Human iPSCs

Research Objective We will utilize human induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) patients for drug testing and drug discovery for this rare genetic disease. Impact Diverse iPSC lines that recapitulate patient phenotypes will supplement preclinical studies to de-risk clinical trials while identifying a therapeutic target for DMD-associated cardiomyopathy. Major Proposed Activities […]

Enabling non-genetic activity-driven maturation of iPSC-derived neurons

Research Objective We will empower stem cell biologists to generate iPSC-derived neurons faster and with enhanced maturation by enabling optical cell stimulation and triggering activity-dependent maturation processes Impact Our project will address such critical bottlenecks as insufficient maturity of iPSC-derived neurons that limits their utility in age-related neurological disorders that manifest later in life. Major […]

A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease

Research Objective We propose to discover a tool that will utilize patient specific iPSC-derived human mini-guts to identify personalized antifibrotic treatments in pediatric Crohn’s disease patients Impact The major bottleneck in intestinal fibrosis research is the difficulty in obtaining patient-specific biologically relevant cells for in vitro modeling. This iPSC-derived tool would overcome it. Major Proposed […]

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