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Developing a gene therapy for IMPG2-Associated Retinal Degeneration in a knockout mouse model

Award Number: F31EY039091
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS
PERIOD OF PERFORMANCE START DATE: 08/01/2026
PERIOD OF PERFORMANCE END DATE: 07/31/2029

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Developing a gene therapy for IMPG2-Associated Retinal Degeneration in a knockout mouse model - Abstract IMPG2-associated retinal degeneration (IMPG2-RD) is an inherited form of progressive vision loss. IMPG2 is a crucial extracellular matrix protein that maintains photoreceptor structure and function, thus deleterious mutations result in photoreceptor cell death. Biallelic mutations in IMPG2 cause a severe, early-onset form of retinal degeneration that includes early macular involvement, while monoallelic mutations result in a later-onset form of IMPG2-RD characterized by macular vitelliform lesions and vision loss that is milder than the biallelic disease. No treatment is currently available for either form of IMPG2-RD. However, here, I propose to engineer a sight-preserving therapy that uses adeno-associated viruses (AAV) to deliver a functional IMPG2 cDNA sequence to photoreceptors. Towards this goal, the Philpot Lab has developed an Impg2-knockout (Impg2-KO) mouse line that recapitulates key features of the human disease and can therefore be a testbed for therapeutic development. Although adult Impg2-KO mice display well-characterized retinal thinning and vitelliform-like subretinal deposits, early disease progression has not yet been studied in detail. Completion of an early timepoint-focused natural history characterization of this mouse model is anticipated to clarify the early cellular dysregulation in IMPG2-RD that precedes photoceptor death and to reveal early biomarkers of disease (Aim 1). Identified early biomarkers can be leveraged to accelerate the evaluation of the proposed viral-mediated gene therapy (Aim 2). Together, these two Aims serve to test my central hypothesis that a gene augmentation approach can preserve retinal structure and function in Impg2-KO mice. This work will provide critical insights into early IMPG2-RD pathology, and in fulfilment of the present unmet need, will support the development of a therapeutic for patients.


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 = $41,404 )
2026	2026	UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL	104 AIRPORT DR STE 2200	CHAPEL HILL	NC	27599	ORANGE	USA	Vision Research	001	1	6/23/2026	NEW	$0
2026	2026	UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL	104 AIRPORT DR STE 2200	CHAPEL HILL	NC	27599	ORANGE	USA	Vision Research	000	1	6/23/2026	NEW	$41,404
														Subtotal = $41,404

Grand Total All Awards = $41,404

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Developing a gene therapy for IMPG2-Associated Retinal Degeneration in a knockout mouse model

Award Number: F31EY039091

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS

PERIOD OF PERFORMANCE START DATE: 08/01/2026

PERIOD OF PERFORMANCE END DATE: 07/31/2029

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