Therapeutic/Technology: Technology
Using metabolic pausing for maintaining stable and high-quality pluripotent stem cells
Autologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cells
Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application
Research Objective Genetically modified cells that help control immune reactions Impact Improved treatment for patients undergoing transplantation Major Proposed Activities To identify the optimal source of regulatory T cells for introduction of the novel IL2 receptor To test the impact of a novel IL2 protein on activating the regulatory T cells To determine the optimal […]
A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering
Research Objective We propose to discover genome- and epigenome-edited allogeneic T cells engineered to selectively target JCV as a potentially lifesaving treatment for progressive multifocal leukoencephalopathy (PML). Impact If successful, we would revolutionize treatment for PML patients in whom T cell immunity cannot be restored or boosted and unable to wait for adoptive transfer of […]
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 […]
Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients
Research Objective We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1 diabetes. Impact Our product will replace donor islets for cell replacement therapy in patients with type 1 diabetes, and will provide a readout of cell […]
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 […]
A novel hybrid CRISPR tool for gene network perturbation and hiPSC engineering
Research Objective A CRISPR-based tool for simultaneous up- and downregulation of many (~5-20) genes, and a computational tool using scRNA-seq data to predict which genes to perturb for efficacious cell-type conversion. Impact A critical bottleneck to the creation of specific cell types from stem cells (and related therapies) is our current inability to make cells […]
iPSCs as a screening tool to predict risk of nonalcoholic fatty liver disease
Research Objective The objective of this proposal is to established undifferentiated iPSCs as a diagnostic tool for the prediction of nonalcoholic fatty liver disease onset and severity. Impact Despite the widespread estimated prevalence of NAFLD, there are currently no tools available to predict likelihood of NAFLD susceptibility beyond standard clinical and demographic information. Major Proposed […]