An IPSC cell based model of macular degeneration for drug discovery.
Research Objective Towards a cure for age related blindness, we propose to create a human stem cell based model to screen for drugs that are protective against Age related macular…
A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease
Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology…
Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies
Research Objective Approximately 20,000 babies are born annually with kidney disease; the long-term outcome is poor. These studies address new ways to develop mini-kidney structures for transplantation to induce repair.…
Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion
Research Objective We aim to develop conditions for stable expansion of blood stem cells outside of the body Impact Blood stem cells are a rare but necessary cell type for…
Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy
Research Objective This study examine potential immunologic changes caused by cellular reprogramming that could present a barrier to clinical application of regenerative therapies. Impact Identification and evaluation of immunologic changes…
Novel metabolic labeling method for tracking stem cells to irradiated salivary glands using PET
Research Objective This project aims to develop a sensitive and non-invasive method for tracking stem cells in clinical trial, without the need for genetically engineered reporters or long-lived radioisotopes. Impact…
Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration
Research Objective We will generate human airway stem cells by direct transcriptional reprogramming of fibroblasts. We will use these induced airway stem cells to model motile cilia disease in a…
Reprogramming human stem cells for blood cell generation
Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create…
Prodrug innovation to target muscle stem cells and enhance muscle regeneration
Research Objective To target therapeutics to muscle stem cells, the building blocks of skeletal muscle. Impact Drugs, genes and gene editing strategies can be delivered directly to muscle stem cells…
Generation of expandable, self-renewing muscle stem cells for Duchenne Muscular Dystrophy
Research Objective The goal of this proposal is to define protocols to generate expandable, self-renewing human muscle stem cells (MuSC) from hiPS cells for Duchenne Muscular Dystrophy disease modeling and…
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