2022
DOI: 10.3390/molecules27020536
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Small Drugs, Huge Impact: The Extraordinary Impact of Antisense Oligonucleotides in Research and Drug Development

Abstract: Antisense oligonucleotides (ASOs) are an increasingly represented class of drugs. These small sequences of nucleotides are designed to precisely target other oligonucleotides, usually RNA species, and are modified to protect them from degradation by nucleases. Their specificity is due to their sequence, so it is possible to target any RNA sequence that is already known. These molecules are very versatile and adaptable given that their sequence and chemistry can be custom manufactured. Based on the chemistry be… Show more

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Cited by 57 publications
(50 citation statements)
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“…RNA interference (RNAi) is effectiveness in transcriptive level as a novel administration. For bacterial elimination, antisense RNA can recruit RNase to degrade base-paired mRNA and repel RNA polymerase to block translation ( Quemener et al, 2022 ). Similarly, miRNA and siRNA can form RNA-induced silencing complex intraocularly for eukaryote, which are enable to modulate bone homeostasis ( Jinek and Doudna, 2009 ).…”
Section: Peptide-based Bone-targeted Therapeutic Strategy Negatively ...mentioning
confidence: 99%
See 2 more Smart Citations
“…RNA interference (RNAi) is effectiveness in transcriptive level as a novel administration. For bacterial elimination, antisense RNA can recruit RNase to degrade base-paired mRNA and repel RNA polymerase to block translation ( Quemener et al, 2022 ). Similarly, miRNA and siRNA can form RNA-induced silencing complex intraocularly for eukaryote, which are enable to modulate bone homeostasis ( Jinek and Doudna, 2009 ).…”
Section: Peptide-based Bone-targeted Therapeutic Strategy Negatively ...mentioning
confidence: 99%
“…Depending on DNA/RNA heteroduplex formation, RNA cleavage is induced by RNase, and the mRNA expression level is ultimately modulated. ASOs are highly valuable as a novel strategy to treat a wide range of diseases linked to dysregulated gene expression ( Quemener et al, 2022 ). The two-component regulatory system (TCS) commonly comprises two components termed “the sensor kinase” and “the response regulator.” TCSs are ubiquitous in bacteria but rare in mammalian cells.…”
Section: Nucleic Acid-based Therapeutic Strategy Inhibits Biofilm For...mentioning
confidence: 99%
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“…The phosphorothioate modification of the backbone greatly improved the binding of the oligonucleotides to the plasma proteins, increasing the time of the oligonucleotides in the circulation, while reducing their elimination. In vivo stability and resistance to degradation by DNases or RNases have been greatly improved by the different chemical modifications introduced in the nucleotides and in the backbone [ 130 , 131 ]. Regarding the delivery to the target cells, oligonucleotides are hydrophilic molecules and cannot passively cross the plasma membrane in the same way as the small molecule lipophilic drugs.…”
Section: Challenges and Solutions For The Clinical Success Of Oligonu...mentioning
confidence: 99%
“…Finally, the third generation ASOs shows the most enhanced resistance against nucleases and the best target affinity, compared to the previous generations [ 144 , 145 , 146 ]. The last generation is also potentially considered the most safe, thanks to its high specificity, affinity, and very limited off-target effects that can be avoided with many bioinformatic tools [ 131 ]. Thus, the chemical modification introduced into DNA or RNA oligonucleotides ( Figure 2 ) have allowed an improvement in the specificity, stability, safety and pharmacokinetic/pharmacodynamic profiles, making this class of compounds more reliable for clinical purposes [ 131 , 144 ].…”
Section: Challenges and Solutions For The Clinical Success Of Oligonu...mentioning
confidence: 99%