CU researchers awarded NEI's 3D ROC prize to accelerate development of new therapies

Natalia Vergara, PhD, assistant professor of ophthalmology at the University of Colorado School of Medicine, has been awarded a 3D ROC prize by the National Eye Institute (NEI) for her research team's work to create better models to accelerate the development of new therapies for retinal diseases.

The prize competition was established by the NEI to promote research on creating improved three-dimensional retinas in vitro, known as retinal organoids, derived from human stem cells,that can help researchers across the country with their work. The full name of the 3D ROC competition is 3D Retina Organoid Challenge.

Vergara and her team were the awardees in Phase II of the NEI's 3D ROC competition, receiving $60,000 for their work developing an organoid model that mimics the composition of the human retina and can respond to light. A key innovation of the team's project was the use of engineered stem cells that allow different cell types in these retinas to fluoresce in different colors, and the combination of this system with a state-of-the-art technology that enables the quantification of those cells in real time. This breakthrough allows for the application of human retinal organoids to the screening and validation of drugs as potential treatments for blinding diseases.

Vergara conducts research on the Anschutz Medical Campus and she is a member of CellSight, a multidisciplinary research initiative that aims to develop stem cell-based therapeutics to save and restore sight in patients with blinding diseases.

In the past decade, the advent of human stem cell-derived retinal organoid models created new opportunities to improve the drug development pipeline by increasing efficiency and decreasing costs. These models make it possible to test drug candidates in three-dimensional human retinal tissues.

The challenge for researchers has been standardizing the process of developing the retinal organoids from induced pluripotent stem cells. Through its work, Vergara's team has been able to create a process for making light responsive retinal organoids that have consistent structure and cellular composition. The researchers' process also improved the yield of retinal organoids and allows researchers to track the cells over a period of time.

Vergara and her fellow CellSight researcher, Valeria Canto-Soler, PhD, describe the research in the video "Improved Fluorescent Reporter Quantification-Based 3D Retinal Organoid Paradigm for Drug Screening." The project is a collaboration with researchers at Miami University, and Nanoscope Technologies.