Gemayel, Michael C.Bhatwadekar, Ashay D.Ciulla, Thomas2024-03-072024-03-072021Gemayel MC, Bhatwadekar AD, Ciulla T. RNA therapeutics for retinal diseases. Expert Opin Biol Ther. 2021;21(5):603-613. doi:10.1080/14712598.2021.1856365https://hdl.handle.net/1805/39095Introduction: In the retina, noncoding RNA (ncRNA) plays an integral role in regulating apoptosis, inflammatory responses, visual perception, and photo-transduction, with altered levels reported in diseased states. Areas covered: MicroRNA (miRNA), a class of ncRNA, regulates post-transcription gene expression through the binding of complementary sites of target messenger RNA (mRNA) with resulting translational repression. Small-interfering RNA (siRNA) is a double-stranded RNA (dsRNA) that regulates gene expression, leading to selective silencing of genes through a process called RNA interference (RNAi). Another form of RNAi involves short hairpin RNA (shRNA). In age-related macular degeneration (AMD) and diabetic retinopathy (DR), miRNA has been implicated in the regulation of angiogenesis, oxidative stress, immune response, and inflammation. Expert opinion: Many RNA-based therapies in development are conveniently administered intravitreally, with the potential for pan-retinal effect. The majority of these RNA therapeutics are synthetic ncRNA's and hold promise for the treatment of AMD, DR, and inherited retinal diseases (IRDs). These RNA-based therapies include siRNA therapy with its high specificity, shRNA to 'knock down' autosomal dominant toxic gain of function-mutated genes, antisense oligonucleotides (ASOs), which can restore splicing defects, and translational read-through inducing drugs (TRIDs) to increase expression of full-length protein from genes with premature stop codons.en-USPublisher PolicyAntisense oligonucleotidesInherited retinal diseaseMicroRNANoncoding RNARNA therapeuticsShort hairpin RNASmall interfering RNATranslational read-through inducing drugsArticle