Gene therapy using adeno-associated viral (AAV) vectors currently represents the most\npromising approach for the treatment of many inherited retinal diseases (IRDs), given AAVâ??s\nability to efficiently deliver therapeutic genes to both photoreceptors and retinal pigment\nepithelium, and their excellent safety and efficacy profiles in humans. However, one of the main\nobstacles to widespread AAV application is their limited packaging capacity, which precludes their\nuse from the treatment of IRDs which are caused by mutations in genes whose coding sequence\nexceeds 5 kb. Therefore, in recent years, considerable effort has been made to identify strategies to\nincrease the transfer capacity of AAV vectors. This review will discuss these new developed\nstrategies, highlighting the advancements as well as the limitations that the field has still to\novercome to finally expand the applicability of AAV vectors to IRDs due to mutations in large\ngenes.
Loading....