Cell Line Generation: iPS Cell


hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders

Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders. Impact This proposal addresses a significant unmet clinical need for a cell therapy approach for gastrointestinal motility disorders such as Hirschsprung disease, achalasia and gastroparesis. […]

Modeling Retinitis Pigmentosa using patient-derived human iPSC organoids

Research Objective The objective of this proposal is to develop a human retinal organoid model of adRP to gain insights in pathogenesis and assess clinically relevant approaches to restore RHO protein function. Impact Upon successful completion of this study, we will have established a disease-in-a-dish model and a novel therapeutic approach towards management of the […]

Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis

Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS disease phenotypes Impact Our goal is to determine if ataxin-3 genetic dysregulation is a target for the development of therapies to treat ALS (Lou Gehrig's […]

Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies

Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]

Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy

Research Objective Using authentic in vitro models of the human lung, complete with inflammatory cells & vessels, we will validate drugs that might be rapidly repurposed for use in patients with COVID-19. Impact The impact will be the avoidance of an animal model once an approved medication hit has been verified by our model. The […]

Identifying a lead compound for COVID19 using high throughput screening with lung stem cell organoids

Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be repurposed for COVID-19 infection. Impact If successful, we will find a therapy to treat COVID-19 infection and prevent the lung complications that are so deadly. […]

Development of a VAV2 antisense oligonucleotide (ASO) treatment for ALS

Research Objective Patient specific stem cells (iPSCs) to model ALS and identify a broadly acting therapeutic intervention Impact Heterogenous patient population, dire need for broadly acting therapeutic interventions Major Proposed Activities Compare the in vitro efficacy of up to 10 lead candidate VAV2 ASOs to rescue the survival of iNs from 30 locally and nationally […]

Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids

Research Objective Our goal is to use stem cell models to identify a small molecule drug candidate for GI motility disorders. Impact The candidate will be used for severe gastrointestinal motility disorders including chronic constipation, achalasia and gastroparesis. Major Proposed Activities Assessment of candidate compounds on stem cell derived models from diverse genetic backgrounds Evaluation […]

Reprogramming Somatic Cells into iPSCs Engineered with an Anti-PSCA CAR to Develop Allogeneic Off-the-Shelf Cell Therapy to Treat Pancreatic Cancer

Research Objective Our candidate product PSCA-CAR_s15 uiNK is derived from transduction of iPSCs selected from the most ideal source and episomally reprogrammed from mature NK cells or CD34+ cells. Impact Cell Functionality and Quality; Scale up and Manufacture Major Proposed Activities Development and characterization of induced pluripotent stem cells (iPSCs) via somatic cell reprogramming of […]

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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