LAUSR

I retinal dystrophies (IRDs) are a rare group of hereditary diseases that lead to progressive degeneration of retinal cells.[1] While there are currently several ongoing clinical trials utilizing pharmacological agents and adeno‐associated virus (AAV) vector‐mediated gene augmentation therapeutics, only one Food and Drug Administration approved therapy currently exists that is merely capable of treating a small fraction of the population: those afflicted by mutations in the RPE65 gene.[2‐7] At this point in time, countless physicians and scientists are poised to address this unmet need for a treatment or cure for IRDs, however, given that most therapies are mutation specific, this task is both highly cost‐ and time‐inefficient.[8] Moreover, we must overcome several crucial obstacles, including on‐ and off‐targeting in genome editing techniques,[9] delivery of genes with a payload too large for that of an AAV vector delivery system,[10] the complexity of removing the gain‐of‐function allele to repair autosomal dominant genes, and a system of ensuring long‐term efficacy of gene augmentation.[11]