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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.
Seminars in Ophthalmology
|May 19, 2021
Summary
Antisense oligonucleotides (AON) are nucleic acid fragments targeting specific mRNA to alter gene products. They show promise for ophthalmic diseases like inherited retinal disorders, offering an alternative to gene therapy.
Area of Science:
- Biotechnology
- Molecular Biology
- Ophthalmology
Background:
- Antisense oligonucleotides (AON) are synthetic nucleic acid fragments designed to bind specific messenger RNA (mRNA) sequences.
- AON have been approved for cytomegalovirus retinitis and show potential for Mendelian systemic diseases.
- Their application is expanding into various ophthalmic conditions, including inherited retinal disorders (IRD).
Purpose of the Study:
- To review the historical development of AON.
- To examine current clinical applications of AON in ophthalmology.
- To discuss ongoing clinical trials involving AON for eye diseases.
Main Methods:
- Review of historical data on AON development.
- Analysis of clinical trial data for AON in ophthalmic diseases.
- Literature review of AON mechanisms and applications.
Main Results:
- AON offer a therapeutic strategy by modifying gene expression at the mRNA level.
- They are being investigated for IRD, glaucoma surgery complications, and macular degeneration.
- AON present a viable gene therapy alternative for IRD cases unsuitable for adeno-associated virus (AAV) delivery.
Conclusions:
- AON represent a significant advancement in nucleic acid-based therapeutics for ophthalmic diseases.
- Ongoing research and clinical trials are crucial for realizing the full potential of AON.
- AON provide a promising therapeutic avenue for complex eye conditions, including genetic disorders.
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