Antisense Oligonucleotides: An Emerging Area in Drug …
Mechanism of action of antisense oligonucleotides (ASOs): ASOs act by either causing (1) RNA cleavage or (2) RNA blockage. (1a) RNase H1 mediated cleavage, (1b) RNA interference (RNAi), (2a) Steric hindrance, and (2b) Splice modulation. (1a) ASO-mRNA heteroduplex recruits RNase H1 enzyme and this enzyme cleaves the target mRNA.
Antisense oligonucleotides: a novel Frontier in pharmacological ...
Mechanism of action of antisense oligonucleotides. Antisense oligonucleotides and siRNA can be used to target a specific, complementary (coding or non-coding) RNA. Main mechanisms of actions are: 1) RNA cleavage through the recruitment of endogenous enzymes; 2) Steric hindrance; 3) Splicing modulation; 4) Activation of RISC complex by the ...
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
2012年1月20日 · Steric-blocking oligonucleotides block the access of cellular machinery to pre-mRNA and mRNA without degrading the RNA. Through this mechanism, steric-blocking...
Antisense oligonucleotide based therapeutics and its …
2023年12月1日 · Mechanism of action of antisense oligonucleotides. Upon entering the cell, antisense oligonucleotide (ASO) mediates its effect in a sequence-dependent manner. The most commonly observed mechanisms include RNase H-mediated cleavage of mRNA or inhibition of protein translation by steric hindrance.
The potential of antisense oligonucleotides in gene silencing was discovered over 40 years ago, which resulted in the growing interest in their chemistry, mechanism of action, and metabolic pathways. This review summarizes the selected mechanisms of antisense drug action, as well as therapeutics which are
Antisense Oligonucleotides and RNA Interference - PMC
Antisense technology presents an opportunity to manipulate gene expression within the cells to treat various diseases, and acts as a powerful tool for studying gene function utilizing antisense agents to manage the diseases by regulating the expression of the specific factor that actually causes the particular disease.
Antisense Oligonucleotides: Basic Concepts and Mechanisms
2002年3月1日 · On the basis of mechanism of action, two classes of antisense oligonucleotide can be discerned: (a) the RNase H-dependent oligonucleotides, which induce the degradation of mRNA; and (b) the steric-blocker oligonucleotides, which physically prevent or inhibit the progression of splicing or the translational machinery.
Cell penetrating peptide conjugates of steric block oligonucleotides
2008年3月1日 · Charge neutral steric block oligonucleotide analogues, such as peptide nucleic acids (PNA) or phosphorodiamidate morpholino oligomers (PMO), have promising biological and pharmacological properties for antisense applications, such …
mechanisms of action. The initial mechanism pertains to the disintegration of RNA, whereas the subsequent mechanism involves the hindrance of RNA through inhibitory or modulatory effects caused by steric factors (Figure 1). ASOs act through two mecha-nisms: degrading targeted RNA sequences and inhibiting translation
Chemistry, mechanism and clinical status of antisense oligonucleotides ...
2017年12月12日 · The two primary antisense approaches for regulating expression through recognition of cellular RNAs are single-stranded antisense oligonucleotides and duplex RNAs. This review will discuss the chemical modifications and molecular mechanisms that make synthetic nucleic acid drugs possible.