Jason Bouna
Research Mentor: Dongshan Yang
Mentor Department: Internal Medicine, Medicine
Author(s): Jason Bouna, Dongshang Yang, Bo Liang
Session: Session 1 (9:00 AM – 9:50 AM)
Presentation Type: Poster 71
Abstract
Retinal gene editing therapies are the gateway to developing cures for Retinitis Pigmentosa (RP), a group of genetic eye diseases that target the retina and destroy photoreceptors that are essential to vision. To develop the cure for RP, a rabbit model of Usher Syndrome Type 3A (USH3A), which presents RP phenotype in the retina, was created. The problem is that USH3A rabbits take years to develop the RP phenotype. This makes developing therapeutics difficult for this model. The aim of this study is to accelerate the progression of RP through exposing USH3A rabbits to strong light intensity. Age matched wild type and USH3A rabbits will endure four hours of high light intensity (800 lux) in a reflective tent four days a week. The visual response to the light exposure will be measured every month using optical coherence tomography (OCT) to scan and visually observe the thickness of the Outer Nuclear Layer (ONL), where photoreceptor nuclei are located, and using electroretinography (ERG) to measure retinal function (response to light). Fundus imaging will be performed to facilitate high-resolution analysis of the retinal surface and underlying vasculature. Through comparative analysis with control groups, we expect to pinpoint the exact window where light-induced USH3A rabbits exhibit profound photoreceptor loss (thinner ONL) and functional ERG impairment. Validating this rapid-induction protocol transforms the USH3A rabbit into a high-throughput model, allowing for the assessment of novel therapeutics within months rather than years and significantly accelerating the translation of life-changing treatments to patients.
