Molecular Therapies for Inherited Retinal Diseases

Following the implementation of next-generation sequencing technologies (e.g. exome and genome sequencing) in molecular diagnostics the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel opportunities to counteract the molecular consequences of these defects are rapidly emerging providing hope for personalized medicine. Classical gene augmentation therapy has been under study for several genetic subtypes of IRD and can be considered a safe and sometimes effective therapeutic strategy. The recent market approval of the first retinal gene augmentation therapy product (LuxturnaTM for individuals with bi-allelic RPE65 mutations) by the FDA has not only demonstrated the potential of this specific approach but also opened avenues for the development of other strategies. However every gene-or even every mutation-may need a tailor-made therapeutic approach in order to obtain the most efficacious strategy with minimal risks associated. In addition to gene augmentation therapy other subtypes of molecular therapy are currently being designed and/or implemented including splice modulation DNA or RNA editing optogenetics and pharmacological modulation. In addition the development of proper delivery vectors has gained strong attention and should not be overlooked when designing and testing a novel therapeutic approach. In this Special Issue we aim to describe the current state of the art of molecular therapeutics for IRD and discuss existing and novel therapeutic strategies from idea to implementation and from bench to bedside.