RNA interference – new prospective direction in drug development

Česká verzia

Authors: Vladimír Soška ^1,2; Ondřej Kyselák ^1,3
Authors place of work: Oddělení klinické biochemie FN U sv. Anny v Brně ¹; II. interní klinika LF MU a FN U sv. Anny v Brně ²; Katedra laboratorních metod LF MU, Brno ³
Published in the journal: AtheroRev 2023; 8(1): 38-44
Category: Reviews

Summary

Biological therapy, whose mechanism of action is the use of monoclonal antibodies against a protein, has been used in clinical practice for many years. However, new drugs from the group of biological therapies that act on the principle of RNA interference are now entering clinical practice. RNA interference is the process by which cells in all living organisms regulate the expression of their genes, and in which the transfer of information about the synthesis of a particular protein between DNA and ribosomes can be stopped. For therapeutic purposes, this effect is achieved by administering artificially synthesized oligonucleotides – short chains of RNA with a precise nucleoside sequence. These are either short stretches of double- stranded RNA or single-stranded oligonucleotides. For clinical use, their chemical modification was necessary to increase their stability and remove some of their side effects, and then binding to other substances to allow their targeted transport to the desired tissue. A number of these drugs are already in advanced stages of clinical trials, and some are entering the pharmaceutical market.

Keywords:

biological therapy – Small interfering RNA – LDL-cholesterol – antisense oligonucleotide – N-acetylgalactosamine – RNA-interference

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