RNA therapeutics in ophthalmology - translation to clinical trials

The use of RNA interference technology has proven to inhibit the expression many target genes involved in underlying pathogenesis several diseases affecting various systems. First established vitro and later animal studies, small interfering (siRNA) antisense oligonucleotide (ASO) therapeutics are now entering clinical trials with potential translation patients. Gene-silencing therapies have demonstrated promising responses ocular disorders, predominantly due structure eye being a closed compartmentalised organ. However, although efficacy such treatments been observed both preclinical studies trials, there issues pertaining these drugs which require more extensive research regards delivery stability siRNAs ASOs. This would improve their for long-term treatment regimens alleviate difficulties experienced by patients diseases. review provides detailed insight into recent developments that conducted gene-silencing therapies, including ISTH0036, SYL040012, SYL1001, PF-04523655, Sirna-027, QR-110, QR-1123, QR-421a IONIS-FB-LRX glaucoma, dry disease, age-related macular degeneration, diabetic oedema inherited retinal Our aim is explore whilst evaluating associated advantages disadvantages, discuss future ophthalmology.