Antisense Oligonucleotide Therapy for Ophthalmic Conditions.
Kevin FerenchakIris DeitchRachel M HuckfeldtPublished in: Seminars in ophthalmology (2021)
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.
Keyphrases
- nucleic acid
- inflammatory response
- optical coherence tomography
- clinical trial
- diabetic retinopathy
- wound healing
- copy number
- genome wide
- minimally invasive
- genome wide identification
- binding protein
- gene therapy
- optic nerve
- coronary artery bypass
- epstein barr virus
- lipopolysaccharide induced
- systematic review
- coronary artery disease
- cataract surgery
- transcription factor
- surgical site infection
- lps induced
- mass spectrometry
- high speed
- immune response
- acute coronary syndrome
- high resolution
- combination therapy
- genome wide analysis
- disease virus