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Antisense therapy - Wikipedia
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 … See more
The common stem for antisense oligonucleotides drugs is -rsen. The substem -virsen designates antiviral antisense oligonucleotides. See more
Half-life and stability
ASO-based drugs employ highly modified, single-stranded chains of synthetic nucleic acids that achieve wide tissue distribution with very long half … See moreCurrent clinical trials
As of 2020 more than 50 antisense oligonucleotides were in clinical trials, including over 25 in See moreAugust 1998Fomivirsen was approved by the U.S. FDA as a treatment for cytomegalovirus retinitis.January 2013Mipomersen was approved by the FDA for the treatment of homozygous familial hypercholesterolemia.December 2016Nusinersen received regulatory approval from FDA and soon after, from other regulatory agencies worldwide.May 2019Volanesorsen was approved by the European Medicines Agency for the treatment of familial chylomicronaemia syndrome.October 2018Inotersen received FDA approval for the treatment of hereditary transthyretin-mediated amyloidosis.2019Milasen was approved by the FDA for the treatment of Batten disease.2020Eteplirsen, golodirsen, and viltolarsen received FDA approval for the treatment of cases that can benefit from skipping exon 51 or 53 of the dystrophin transcript.2023Tofersen was approved by the FDA for the treatment of SOD1- associated amyotrophic lateral sclerosis.Amyotrophic lateral sclerosis
Tofersen (marketed as Qalsody) was approved by the FDA for the treatment of SOD1- associated amyotrophic lateral sclerosis See moreWikipedia text under CC-BY-SA license Antisense RNA - Wikipedia
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WEBFeb 12, 2019 · Abstract. Nucleic acids are the backbone of antisense therapy. Antisense oligonucleotide-based therapeutics involves downregulation of gene expression.
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WEBMay 20, 2005 · Key Points. Antisense oligonucleotides (ASOs) offer one approach to target genes involved in cancer progression, particularly those that are not amenable to small-molecule or antibody inhibition....
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The powerful world of antisense oligonucleotides: …
WEBMar 31, 2020 · Antisense oligonucleotides (ASOs) represent a new and highly promising class of drugs for personalized medicine. In the last decade, major chemical developments and improvements of the …
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