A modified arrestin1 to enhance glycolysis in photoreceptors as a therapeutic approach to slowing retinal degeneration

NIH RePORTER · NIH · R01 · $391,460 · view on reporter.nih.gov ↗

Abstract

SUMMARY/ABSTRACT There are currently more than 280 genes in which defects are known to cause retinal degeneration. Thus, there is a critical need to develop broad spectrum approaches to treating these diseases. The hypothesis of this proposal is that if we can selectively increase glycolysis in rod and cone photoreceptors, then this increase in metabolic potential should slow retinal degeneration across a broad spectrum of etiologies. Our findings strongly support the idea that we can specifically modify arrestin1 such that the catalytic rate of enolase1 in glycolysis is selectively increased in rods and cones. Significantly, this increase in glycolysis slows the loss of photoreceptors and improves photoreceptor function in at least one animal model of retinal degeneration. Accordingly, the specific aims of this proposal are to determine the mechanistic properties of ArrGG’s effect, examining metabolites and gene expression profiles of treated animals (Aim1). We will then establish ArrGG as a gene-agnostic approach to slowing retinal degeneration, testing the therapeutic benefit of ArrGG in diverse models of inherited retinal degeneration (Aim 2).

Key facts

NIH application ID
10782168
Project number
1R01EY035768-01
Recipient
UNIVERSITY OF FLORIDA
Principal Investigator
W CLAY SMITH
Activity code
R01
Funding institute
NIH
Fiscal year
2024
Award amount
$391,460
Award type
1
Project period
2024-01-01 → 2028-12-31