Part of the book: Drug Development
Part of the book: Ocular Diseases
RNA interference (RNAi) is a posttranscriptional mechanism of gene regulation present in eukaryotic cells. Inducers of RNAi are small molecules of RNA that act in the cytoplasm where they are able to impair translation of a specific mRNA to protein, hence modifying gene expression. The discovery of this mechanism in mammals led to the development of a new class of therapeutics with the aim of exploiting this endogenous mechanism of action. In the last decade, great efforts have been put into understanding RNAi and translating this accumulated knowledge into the design of modern therapeutics. With several compounds in phase III clinical development, the field is getting closer to its first market authorization. Here we make a thorough overview of the field of RNAi therapeutics in ophthalmology, one of the fields in which RNAi has been most successful.
Part of the book: RNA Interference
Primary open-angle glaucoma is a progressive ocular neuropathy that if left untreated may lead to blindness. The main risk factor for developing glaucoma is increased intraocular pressure. Intraocular pressure is regulated by the balance of aqueous humour synthesis and secretion into the eye and outflow from the eye; therefore, most therapies for glaucoma seek lowering intraocular pressure to avoid disease progression. There are several types of drugs in the market for the treatment of glaucoma, but there are still unmet needs to be overcome; therefore, significant effort has been put in the last few years to develop new medicines with innovative mechanisms of action as well as devices to improve quality of life in glaucoma patients. The present review offers a thorough revision of the latest advances in the glaucoma therapy field, focusing on innovative approaches, new targets and new mechanisms of action.
Part of the book: Glaucoma