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Autologous transplantation of genetically modified induced pluripotent stem cells for rescue of autosomal dominant retinal disorders

Award Number: F31EY037576
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS
PERIOD OF PERFORMANCE START DATE: 12/01/2026
PERIOD OF PERFORMANCE END DATE: 11/30/2027

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Autologous transplantation of genetically modified induced pluripotent stem cells for rescue of autosomal dominant retinal disorders - PROJECT SUMMARY Gene therapy for the treatment of autosomal recessive disorders by supplementation with wild-type (WT) copies of the affected gene has been tested in FDA-approved trials. However, strategies for treating autosomal dominant (ad) disorders, which require the removal of a gain-of-function allele through precise genomic repair, have been tested preclinically but have not been evaluated in human subjects. In this proposal, we aim to overcome long standing obstacles to the treatment of RPE65 D477G dominant disorders using autologous induced pluripotent stem cell (iPSC) transplantation. The central hypothesis is that patient-derived iPSC RPE (iRPE) grafts repaired by CRISPR in vitro are a therapeutic option to treat RPE disorders. We will use allele- specific CRISPR-edited RPE65 D477G iRPE to confirm functional repair and differentiation in Aim 1a, and the genomic integrity will be validated via WGS in Aim 1b. The therapeutic potential of iRPE transplantation will be assessed when transplanted into a retinitis pigmentosa preclinical mouse model, Rpe65rd12;Prdcscid. By comparing phenotypes induced by transplantation of repaired iRPE vs. control RPE lines, we will assess whether in vitro CRISPR-repaired iRPE halts disease development in the context of an ad disorder. Our objective is to identify ways to leverage patient stem cells as a tissue source for regenerative medicine. Aim 2 will determine whether patient iRPE repaired in vitro functionally integrate into the retinal circuitry of live mice and rescue vision. Lastly, Aim 3 will determine whether transplanted iRPE is safe and nontumorigenic through long-term analysis.


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 = $50,114 )
2026	2026	THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	615 W 131ST ST	NEW YORK	NY	10027	NEW YORK	USA	Vision Research	000	1	4/13/2026	NEW	$50,114
														Subtotal = $50,114

Grand Total All Awards = $50,114

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Autologous transplantation of genetically modified induced pluripotent stem cells for rescue of autosomal dominant retinal disorders

Award Number: F31EY037576

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS

PERIOD OF PERFORMANCE START DATE: 12/01/2026

PERIOD OF PERFORMANCE END DATE: 11/30/2027

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