New Treatments for Stargardt Disease and Related Retinal Degenerative Diseases.

Abstract

Stargardt disease is a progressive hereditary retinal disease which is currently incurable. Although ABCA4 has been identified as a major causative gene, patients with genetic mutations in other genes (PRPH2, ELOVL4, and PROM1) display similar clinical phenotypes as Stargardt disease. Recent advances in genetic and molecular studies have greatly enhanced our understanding of the disease and have led to identification of targetable pathophysiological progresses and new molecular clinical applications. Here we summarize the current understanding of the pathophysiology and potential therapies for Stargardt disease and other related retinal degenerative diseases.