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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]
The use of Morpholino antisense oligonucleotides for gene knockdowns in vertebrates, which is now a standard technique in developmental biology and is used to study altered gene expression and gene function, was first developed by Janet Heasman using Xenopus. [13] FDA-approved Morpholino drugs include eteplirsen and golodirsen. The antisense ...
Following is a list of antipsychotics, sorted by class. Antipsychotics. Antipsychotics by class Generic name Brand names Chemical class ATC code
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 ...
A straightforward uptake, like for most small-molecule drugs, is hindered by the polyanionic backbone and the molecular size of ONs. Being adapted from the broad and successful class of Antibody-Drug conjugates , antibodies and antibody analogues are more and more used in research in order to overcome hurdles related to delivery and ...
The drug functions as an antisense oligonucleotide and binds to the complementary sequence of the mRNA that inhibits the replication of human cytomegalovirus. This therapy can also be categorized as Antisense oligonucleotide (ASO) therapy. [32]
Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. [3] It is a transthyretin-directed antisense oligonucleotide. [3]
The US list price of eteplirsen is US$300,000 per year of treatment citation needed. The Institute for Clinical and Economic Review has found the drug not cost effective at the list price when the cost of one Quality-adjusted life year (QALY) was equal to US$150,000. [20]