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.…
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…
Organoid Modeling of Human Cortical Microcircuits
Research Objective The proposed studies will develop three-dimensional cell culture methods for creating human brain neural circuits for disease research and drug discovery. Impact The proposed research will develop a…
Blood Brain Barrier (BBB)-on-Chip: Development and validation of a novel iPS-based microfluidic model of the human BBB
Research Objective to develop and systematically characterize a novel model of the human BBB using a microfluidic device (chip) and cells derived from induced pluripotent stem cells (iPSCs). Impact The…
Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells
Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how…
Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug Discovery
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical…
Human iPSC-derived micro-heart muscles for high-throughput cardiac drug discovery
Translational Candidate In vitro miniaturized array of heart muscle amenable for use in efficient high-throughput drug discovery and screening campaigns. Area of Impact Effective high-throughput screening of drugs on human…
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