Shared Resources Laboratory for Stem Cell-Based Modeling in Stem Cell Biology and Engineering
We propose to expand and enhance our stem cell core, a key resource for the local area.This project will accelerate new therapies and train stem cell scientists. We propose to…
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…
Chemically engineered photoreceptors for vision restoration in retinal degeneration associated blindness.
Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…
Development of an Optogenetic Vision Restoration Gene Therapy Using an Engineered Form of Melanopsin
Research Objective The objective of this research proposal is to develop a lead AAV candidate for an optogenetic vision restoration therapy for patients suffering from blindness due to loss of…
Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease models
Research Objective We will develop a gene therapy for a major inherited optic nerve disease and test the effectiveness of the treatment by analyzing healthy and patient stem cell-derived mini…
Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases
Research Objective We will discover small molecule compounds that correct disease in eyecups (retinal organoids) differentiated from patient iPSCs with photoreceptor diseases. Impact Our small molecule agents will provide new…
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 Novel Tissue Engineering Technique to Repair Degenerated Retina
Research Objective Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases Impact Based on…
Embryonic Stem Cells for Corneal Endothelial Degeneration
Research Objective The proposed studies will determine the optimal approaches to differentiate and transplant stem cell-derived corneal endothelial cells. Impact These data will provide foundational proof-of-concept data that will allow…
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