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Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies

Award Number: R01EY038076
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/01/2026
PERIOD OF PERFORMANCE END DATE: 07/31/2030

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Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies - 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.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $689,706 )
2026	2026	UNIVERSITY OF WISCONSIN SYSTEM	21 N PARK ST STE 6301	MADISON	WI	53715	DANE	USA	Vision Research	000	1	8/4/2026	NEW	$689,706
														Subtotal = $689,706

Grand Total All Awards = $689,706

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Establishing human stem cell derived-retinal organoids as functional models of human retina for studying disease and testing therapies

Award Number: R01EY038076

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 08/01/2026

PERIOD OF PERFORMANCE END DATE: 07/31/2030

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