# Therapeutic Editing of Rod Glycolysis Rescues Retinal Degeneration

> **NIH NIH F31** · COLUMBIA UNIVERSITY HEALTH SCIENCES · 2024 · $48,974

## Abstract

PROJECT SUMMARY
Retinitis pigmentosa (RP) is the most common inherited retinal dystrophy, caused by more than 3100 mutations
in 80 genes that are primarily specific to rod photoreceptors. Following the major rod death phase, cone death
occurs regardless of the underlying gene mutations. However, there exists a knowledge gap in understanding
how aerobic glycolysis in cones impacts the delicate “metabolic coupling” between cones and retinal pigment
epithelium (RPE) in RP. In this proposal, we hypothesize that reprogramming rod and cone aerobic glycolysis
can promote cone survival in RP independent of the underlying rod-specific gene mutations. To test this
hypothesis, we propose the following specific aims. In Aim 1, we will determine whether enhanced aerobic
glycolysis in cone photoreceptors can promote their survival in a novel genetic mouse model. In Aim 2, we will
test the potential of gene therapy to slow photoreceptor degeneration by enhancing aerobic glycolysis in mouse
models of RP and characterizing the metabolic changes that occur. Lastly, in Aim 3, we will evaluate the safety
profile of the gene therapy described in Aim 2.

## Key facts

- **NIH application ID:** 10774254
- **Project number:** 5F31EY033660-02
- **Recipient organization:** COLUMBIA UNIVERSITY HEALTH SCIENCES
- **Principal Investigator:** Nicholas David Nolan
- **Activity code:** F31 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $48,974
- **Award type:** 5
- **Project period:** 2023-02-01 → 2026-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10774254

## Citation

> US National Institutes of Health, RePORTER application 10774254, Therapeutic Editing of Rod Glycolysis Rescues Retinal Degeneration (5F31EY033660-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10774254. Licensed CC0.

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