Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies
About This Grant
PROJECT SUMMARY/ABSTRACT Damage and dysfunction of photoreceptors in the retina is one of the leading causes of vision loss and there are currently no treatment options for such retinal degenerative diseases. With the recent breakthroughs in human stem cell technology, it has become possible to recreate a 3D mini retina in a dish called retinal organoids (ROs) from human pluripotent stem cells (hPSC) that share several similarities with human retina including generating electrical signals to light stimuli. However, there are several limitations that remain unidentified or poorly understood which precludes ROs as a robust in vitro model of human retina for studying diseases, testing therapeutics and as a renewable source of human photoreceptors for stem cell replacement therapies. Our goal in this proposal is to determine the limitations in ROs with respect to cone photoreceptor signaling both at the level of signal transduction as well as signal transmission across the photoreceptor synapse to the remaining neural circuitry. In addition, we will also identify the limitations of ROs as a viable alternative for modeling retinal diseases in a dish with the goal of testing gene therapy in a dose-dependent manner. In Aim 1 we will determine the relative contributions of cone photoreceptor-extrinsic, cone photoreceptor-intrinsic as well as developmental factors that limit a uniform population of functional cones with robust phototransduction profiles. In Aim 2 we will delineate the extent to which there is reliable transmission of electrical signaling between cone photoreceptors and the second-order neurons in ROs using a combinatorial approach of anatomical, electrophysiological, and genetic tools. In Aim 3, we will leverage patient-derived ROs and gene-edited ROs to model two distinct retinal diseases that affect cone signaling and use a viral-mediated delivery approach to quantify a dose-response potency assay for gene therapy. Together these aims will help establish ROs as a powerful in vitro model of human retina and as a robust platform for modeling retinal diseases and testing gene therapy.
Grant Summary
Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies is a NEI - National Eye Institute grant providing up to $690K for university, nonprofit, healthcare org. Applications are due 2030-07-31 (open). Check eligibility and apply with FindGrants.
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Up to $690K
2030-07-31
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Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies: Frequently Asked Questions
Who is eligible for the Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies?
Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies is offered by NEI - National Eye Institute and is generally open to university, nonprofit, healthcare org. It is open to organizations nationwide unless the funder specifies otherwise. Review the specific eligibility terms before applying, since funders set their own requirements around organization type, location, and the population or project being served.
How much funding does the Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies provide?
Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies provides up to $690K per award from NEI - National Eye Institute. Actual award sizes depend on the scope of your project, available program funds, and the number of applicants, so build a budget that reflects realistic, allowable costs rather than the maximum figure.
When is the Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies deadline?
Applications for Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies are due 2030-07-31 (open). Because deadlines can change, verify the date with the funder, NEI - National Eye Institute, and give yourself enough time to prepare a complete, competitive application before the close date.
How do you apply for the Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies?
To apply for Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies, confirm your eligibility, gather the required documents, and prepare a narrative and budget that address the funder's priorities. FindGrants guides you step by step and can draft each section, then exports a submission-ready application pack for this grant from NEI - National Eye Institute.