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.…
Research Objective Understanding alveolar progenitor cell defects in T21 and the genes/pathways associated with them will allow for developing therapeutic approaches for individuals with DS. Impact Although trisomy 21 affects…
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…
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 Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
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…
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