Dutch researchers are developing an RNA therapy to target the gene responsible for vision loss in Stargardt disease.
Leading the research into the ABCA4 gene (mutations in which ultimately cause Stargardt disease’s lipofuscin build-up in the retinal pigment epithelium) are Radboud University Centre’s Professor Carel Hoyng and Professor Rob Collin. Speaking at Bascom Palmer Eye Institute’s Angiogenesis, Exudation and Degeneration 2022 virtual conference, Prof Hoyng described antisense oligonucleotides (AONs) as synthetic RNA molecules, delivered intravitreally. AONs restore the proteins normally encoded by the intact ABCA4 gene, targeting the root cause of the condition. Prof Hoyng added that a similar strategy has shown promise in treating Leber congenital amaurosis.
