Abstract
Nucleic acid therapeutics is a growing field aiming to treat human conditions that has gained special attention due to the successful development of mRNA vaccines against SARS-CoV-2. Another type of nucleic acid therapeutics is antisense oligonucleotides, versatile tools that can be used in multiple ways to target pre-mRNA and mRNA. While some years ago these molecules were just considered a useful research tool and a curiosity in the clinical market, this has rapidly changed. These molecules are promising strategies for personalized treatments for rare genetic diseases and they are in development for very common disorders too. In this chapter, we provide a brief description of the different mechanisms of action of these RNA therapeutic molecules, with clear examples at preclinical and clinical stages.
Highlights
Nucleic acid therapeutics is still a growing field
With the irruption of the mRNA vaccines against SARS-CoV-2 special attention has been given to this type of therapies but other types of nucleic acid therapeutics, coined antisense oligonucleotides (AONs), have been studied for many years
A seemingly opposite mechanism of action is at the core of nusinersen, an AON approved for the treatment of spinal muscular atrophy (SMA)
Summary
Nucleic acid therapeutics is still a growing field. With the irruption of the mRNA vaccines against SARS-CoV-2 special attention has been given to this type of therapies but other types of nucleic acid therapeutics, coined antisense oligonucleotides (AONs), have been studied for many years. Only a dozen therapeutic oligonucleotides have been formally approved for clinical use, there are many new such drugs in the pipeline for a plethora of (mainly rare) diseases. These AON molecules interact with different nucleic acids (mRNA, non-coding RNA, and DNA) thanks to sequence specific Watson–Crick base pairing. Their mechanism of action, that may be designed to bind specific targets, makes these drugs easy to design, less likely to cause side effects and, potential candidates to lead the wave of precision medicine. We describe the most frequently used AON-based therapeutic strategies, their mechanisms of action (Fig. 1), and the results of several clinical trials, with special emphasis in eye and muscle diseases
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