Preclinical Development of RNAi-Inducing Oligonucleotide Therapeutics for Eye Diseases
( RNA Interference )
Publication series ： RNA Interference
Author： Tamara Martínez Maria Victoria González Beatriz Vargas AnaIsabel Jiménez and Covadonga Pañeda
Publisher： IntechOpen
Publication year： 2016
E-ISBN: INT6063161803
P-ISBN(Paperback): 9789535122722
P-ISBN(Hardback): 9789535122722
Subject： R394 medical genetics
Keyword： 医学遗传学
Language： ENG
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Preclinical Development of RNAi-Inducing Oligonucleotide Therapeutics for Eye Diseases
Description
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.
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