The development and technologies of RNA therapeutics

Huong Lan Vuong, Chu Thanh Lan, Hien Thi Thu Le

Research output: Chapter in Book/Report/Conference proceedingChapterScientificpeer-review

Abstract

Since it was discovered for over 20 years ago, the potentiality of siRNAs in gene silencing in vitro and in vivo models has been recognized. Several studies in the new generation, molecular mechanisms, target attachment, and purification of RNA have supported the development of RNA therapeutics for a variety of applications. RNA therapeutics are growing rapidly with various platforms contributing to the standard of personalized medicine and rare disease treatment. Therefore, understanding the development and technologies of RNA therapeutics becomes a crucial point for new drug generation. Here, the primary purpose of this review is to provide a general view of six therapeutic categories that make up RNA-based therapeutic approaches, including RNA-target therapeutics, protein-targeted therapeutics, cellular reprogramming and tissues engineering, RNA-based protein replacement therapeutics, RNA-based genome editing, and RNA-based immunotherapies based on non-coding RNAs and coding RNA. Furthermore, we present an overview of the RNA strategies regarding viral approaches and nonviral approaches in designing a new generation of RNA technologies. The advantages and challenges of using RNA therapeutics are also discussed along with various approaches for RNA delivery. Therefore, this review is designed to provide updated reference evidence of RNA therapeutics in the battle against rare or difficult-to-treat diseases for researchers in this field.

Original languageEnglish
Title of host publicationProgress in Molecular Biology and Translational Science
Subtitle of host publicationRNA Therapeutics Part A
EditorsChu Dinh-Toi, Van Thai Than
PublisherElsevier
ChapterII
Pages13-39
Volume203
DOIs
Publication statusPublished - 2024
Publication typeA3 Book chapter

Publication series

NameProgress in Molecular Biology and Translational Science
ISSN (Print)1877-1173
ISSN (Electronic)1878-0814

Keywords

  • Coding RNA
  • MRNA therapeutics
  • Non-coding RNA
  • RNA delivery
  • Viral approach, non-viral approach

Publication forum classification

  • Publication forum level 1

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology

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