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siRNA in neuroretinal disease: from dream to reality
Author(s) -
Levin Leonard A.
Publication year - 2019
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2019.8211
Subject(s) - gene silencing , messenger rna , small interfering rna , translation (biology) , dream , computational biology , disease , microbiology and biotechnology , medicine , biology , rna , bioinformatics , gene , genetics , neuroscience , pathology
Small interfering ribonucleic acid (siRNA) molecules are used for gene silencing by activating a degradation pathway for transcribed mRNA molecules after they are transcribed. By complementing a highly specific sequence of the mRNA, the siRNA prevent translation of the mRNA into the targeted protein. They have long been the subject of translational research as treatments for various diseases, but only recently has the expectation come closer to realization. This talk will discuss the development of improved siRNA therapies and review their use in the laboratory and some recent and currently active clinical trials.