Theoretical and Natural Science

- The Open Access Proceedings Series for Conferences

Theoretical and Natural Science

Vol. 6, 03 August 2023

Open Access | Article

RNA interference: from target validation to therapeutics

Wenda Zhu * 1
1 Southeast University

* Author to whom correspondence should be addressed.

Theoretical and Natural Science, Vol. 6, 57-64
Published 03 August 2023. © 2023 The Author(s). Published by EWA Publishing
This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Citation Wenda Zhu. RNA interference: from target validation to therapeutics. TNS (2023) Vol. 6: 57-64. DOI: 10.54254/2753-8818/6/20230167.


Generally, a new targeted medicine takes a long time, usually 5 to 15 years, from the discovery and validation of its target to its actual use in treating a disease. There is no doubt that steps such as target validation and target evaluation are essential in preclinical development of new drugs to ensure safety, bioavailability, and efficacy of the drug, and in the target validation phase, RNA interference (RNAi) is a very important method. RNAi drug has double-stranded RNA function, so the targeted mRNA of a specific gene sequence is degraded, its transcription process is abnormal, which leads to gene silencing, and related protein synthesis failure. RNAi technology could specifically shut off specific genes, and, it is a targeted drug with great potential. Therefore, RNAi technology can be used to treat some rare diseases, such as acute hepatic porphyria (AHP) in adults, by using drugs as carriers. In 1998, Andrew Fire and Craig C. Mello first defined the RNAi phenomenon and was honored with the Nobel Prize in Physiology or Medicine in 2006 for their discovery of RNAi and their outstanding contributions to the field of gene silencing. Since then, RNAi drugs have been studied by pharmaceutical scientists around the world, but many of the previous drugs have been stopped at the evaluation stage due to their chemical instability. Until October 2018, the world’s first RNAi drug (patisiran) was successfully marketed so that the RNAi drug development became popular again because of remarkable pesticide effect.


RNAi, target validation, assessment, siRNA, miRNA, dsRNA, screening of targets, siRNA-based drug


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Data Availability

The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.

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Volume Title
Proceedings of the International Conference on Modern Medicine and Global Health (ICMMGH 2023)
ISBN (Print)
ISBN (Online)
Published Date
03 August 2023
Theoretical and Natural Science
ISSN (Print)
ISSN (Online)
03 August 2023
Open Access
This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited

Copyright © 2023 EWA Publishing. Unless Otherwise Stated