siRNA in neuroretinal disease: from dream to reality

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...

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Bibliographic Details
Published in:Acta ophthalmologica (Oxford, England) England), 2019-12, Vol.97 (S263), p.n/a
Main Author: Levin, Leonard A.
Format: Article
Language:English
Subjects:
Clinical trials
Gene silencing
Medical treatment
mRNA
siRNA
Online Access:Get full text
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Summary: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.
ISSN:1755-375X
1755-3768
DOI:10.1111/j.1755-3768.2019.8211