Antisense Oligonucleotide Therapy for Ophthalmic Conditions

Antisense oligonucleotides (AON) are synthetic single-stranded fragments of nucleic acids that bind to a specific complementary messenger RNA (mRNA) sequence and change the final gene product. AON were initially approved for treating cytomegalovirus retinitis and have shown promise in treating Mendelian systemic disease. AON are currently being investigated as a treatment modality for many ophthalmic diseases, including inherited retinal disorders (IRD), inflammatory response and wound healing after glaucoma surgery, and macular degeneration. They provide a possible solution to gene therapy for IRD that are not candidates for adeno-associated virus (AAV) delivery. This chapter outlines the historical background of AON and reviews clinical applications and ongoing clinical trials.

Automated summary

Antisense oligonucleotides (AON) are synthetic single-stranded fragments of nucleic acids that bind to specific mRNA sequences and hold promise in treating various ophthalmic diseases, serving as a potential gene therapy solution for IRD that are not suitable for AAV delivery.