UCSF’s Yao Tong, MD, PhD, Receives $90,000 KTEF Grant for Stargardt Disease Research

Yao Tong, MD, PhD, from the University of California San Francisco, was awarded a $90,000 grant for Mechanisms that Drive Mitochondrial Dysfunction in the Retinal Pigment Epithelium in Stargardt Inherited Macular Degeneration.

Stargardt disease (STGD1) is a genetic eye disease that leads to vision loss due to the mutation in the ABCA4 gene in ~1 in 8,000 children. ABCA4 is responsible for preventing the buildup of toxic metabolites of vitamin A in the retina, which harm the retinal pigment epithelium (RPE), the tissue that nourishes and supports the light-sensing photoreceptors. ABCA4 mutations lead to the accumulation of these toxic metabolites in the RPE, leading to the death photoreceptors, and resulting in a gradual and irreversible loss of vision in both children and adults. Unfortunately, there are currently no approved treatments to prevent or halt the loss of vision in Stargardt disease.

The RPE performs numerous functions to support the health of the photoreceptors and relies on its mitochondria to provide energy to power these functions. Dr. Tong has observed damaged dysfunctional mitochondria in Stargardt disease RPE. Insufficient energy impairs the functions of RPE, leading to disease onset.

Dr. Tong proposes to investigate how mitochondria in the RPE are damaged and identify strategies to restore RPE mitochondrial health by targeting specific mechanisms. She anticipates that these studies will provide important insight into mechanisms that drive Stargardt disease and find ways to treat this currently untreatable condition.