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Updated: Nov 5, 2025

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
Published on: March 16, 2022
Antisense Oligonucleotide Therapy for Ophthalmic Conditions
Kevin Ferenchak1, Iris Deitch1, Rachel Huckfeldt1
1Inherited Retinal Disorders Service, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, United States.
Abstract:
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.
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