UCLA’s Elliot Choi, MD, PhD, Receives $100,000 KTEF Grant for Gene-Editing Research in Stargardt Disease

Elliot Choi, MD, PhD from UCLA Jules Stein Eye Institute, Los Angeles, CA, was awarded a $100,000 grant for Multiplex Prime-Editing Strategies for ABCA4 Mutations in Stargardt Disease.

Stargardt disease (STGD1) is the most common inherited cause of vision loss in children and young adults. It leads to progressive damage to the light-sensing cells in the retina. STGD1 is caused by harmful changes in a gene called ABCA4, which is essential for removing toxic byproducts that form during normal vision. When ABCA4 does not function properly, these toxic molecules build up and gradually damage the retina. Although STGD1 affects thousands of children and adolescents in the United States, there are currently no approved treatments that can slow or prevent the disease.

Dr. Choi aims to develop a new therapeutic strategy that corrects the underlying genetic variants responsible for STGD1. Using a highly precise gene-editing tool known as a “prime editor,” he will repair the disease-causing mutations directly within the cells of the retina. He will deliver this DNA-editor into the eye using safe viral or virus-like particles and evaluate its ability to correct the mutation and improve retinal health in a well-established mouse model of STGD1 that carries the same genetic changes that occur in human patients.

Upon successful completion, Dr. Choi will provide the first evidence that prime editing can be used as a treatment for pediatric STGD1. More broadly, it will lay the foundation for developing similar genetic therapies for many other childhood eye diseases caused by known mutations. Ultimately, this research has the potential to preserve vision and improve quality of life for affected children and their families.