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Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
AsRNA is transcribed from the lagging strand of a gene and is complementary to a specific mRNA or sense transcript. Antisense RNA (asRNA), also referred to as antisense transcript, [1] natural antisense transcript (NAT) [2] [3] [4] or antisense oligonucleotide, [5] is a single stranded RNA that is complementary to a protein coding messenger RNA (mRNA) with which it hybridizes, and thereby ...
Eteplirsen's proposed mechanism of action is to bind to dystrophin pre-mRNA and alter the exon splicing of the RNA so that more almost full-length dystrophin is made. By increasing the quantity of an abnormal, but potentially functional, dystrophin protein, the objective is to slow or prevent the progression of DMD.
Antisense oligonucleotides can be used to target a specific, complementary (coding or non-coding) RNA. If binding takes place this hybrid can be degraded by the enzyme RNase H. [12] RNase H is an enzyme that hydrolyzes RNA, and when used in an antisense oligonucleotide application results in 80-95% down-regulation of mRNA expression. [6]
The mechanism of therapeutic gene-silencing action relies on degradation through the action of RNase H. [1] [5] Nearly all organisms utilize this family of enzymes to degrade DNA-RNA hybrids as a defense against viral infection. [6] In protein synthesis, DNA is first transcribed into mRNA, and then translated in an amino acid sequence.
Aganirsen is a 25-mer (5'-TATCCGGAGGGCTCGCCATGCTGCT-3'), first-generation antisense oligonucleotide (phosphorothioate linkage). Aganirsen has a molecular mass of 8036 (Da), a melting point of approximately 64.2 °C, and a GC content of 64%. Aganirsen is highly soluble in water.
Antagomirs, also known as anti-miRs, are a class of chemically engineered oligonucleotides designed to silence endogenous microRNAs (also known as miRNAs or miRs). [1] [2] [3] Antagomirs are a kind of antisense oligonucleotide, as their sequence is complementary to their specific miRNA target. Their structure has modifications so as to make ...
Nusinersen is an antisense oligonucleotide in which the 2'-hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. [4] [11] [12]
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