Types of Rna Therapeutics Publisher Pubmed



Goleij P^{1, 2} ; Babamohamadi M^{2, 3, 4} ; Rezaee A⁵ ; Sanaye PM⁶ ; Tabari MAK^{7, 8} ; Sadreddini S⁹ ; Arefnezhad R^{10, 11} ; Motedayyen H¹²
Source: Progress in Molecular Biology and Translational Science Published:2024

Abstract

RNA therapy is one of the new treatments using small RNA molecules to target and regulate gene expression. It involves the application of synthetic or modified RNA molecules to inhibit the expression of disease-causing genes specifically. In other words, it silences genes and suppresses the transcription process. The main theory behind RNA therapy is that RNA molecules can prevent the translation into proteins by binding to specific messenger RNA (mRNA) molecules. By targeting disease-related mRNA molecules, RNA therapy can effectively silence or reduce the development of harmful proteins. There are different types of RNA molecules used in therapy, including small interfering RNAs (siRNAs), microRNAs (miRNAs), aptamer, ribozyme, and antisense oligonucleotides (ASOs). These molecules are designed to complement specific mRNA sequences, allowing them to bind and degrade the targeted mRNA or prevent its translation into protein. Nanotechnology is also highlighted to increase the efficacy of RNA-based drugs. In this chapter, while examining various methods of RNA therapy, we discuss the advantages and challenges of each. © 2024