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…
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…
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…
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…
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…
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…
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…
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…
Research Objective Develop stem cell-based therapy to treat dementia Impact There are no treatments for dementia. If successfully achieved, this study will lead to a cure of a familial form…
Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum…
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