Date

Friday 23 Oct 2026

Time

09:45 - 10:15

Location

BW020

Supervisor

Rene Olsthoorn

Inherited retinal diseases comprise a genetically heterogeneous group of disorders in which pathogenic variants disrupt the function and survival of retinal cells. Although gene therapy has demonstrated clear clinical potential, the large number of causative genes and differences in disease mechanism and stage make a single therapeutic approach unsuitable for all patients. This review compares four major molecular strategies for inherited retinal diseases: gene augmentation, genome editing, RNA based therapy, and gene agnostic optogenetic therapy. Gene augmentation currently has the strongest clinical validation and is particularly suitable for loss of function disorders in which viable target cells remain. Genome editing enables direct modification of the endogenous genetic defect and offers the possibility of potentially permanent correction, although mutation specificity, delivery, and long-term genomic safety remain important challenges. RNA based therapies provide a non-permanent approach to modify transcript processing or RNA sequence, but their clinical development has highlighted challenges related to durability, delivery, the potential need for repeated administration, and variable efficacy. Optogenetic therapy differs fundamentally by bypassing the original genetic defect and introducing light sensitivity into surviving inner retinal neurons, making it particularly relevant for advanced retinal degeneration. Comparison of these strategies indicates that therapeutic suitability depends not only on genotype but also on molecular disease mechanism, residual retinal structure, and stage of degeneration. The future of inherited retinal disease therapy is therefore likely to rely on a complementary molecular toolbox in which therapeutic strategies are matched to the biological and clinical characteristics of individual patients.