Design of a novel antisense oligonucleotide therapy for inherited blindness

Inherited retinal diseases are a major cause of vision loss for which precision medicine is entering the clinic. Emerging antisense oligonucleotide (ASO) therapies are mostly mutation-specific, requiring many individual clinical trials. Here, we aim to design an innovative, mutation-independent ASO strategy targeting upstream open reading frames in order to increase protein translation, which is potentially applicable for many inherited diseases.