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Antisense therapy information


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] Several ASOs have been approved in the United States, the European Union, and elsewhere.

  1. ^ Morcos PA (June 2007). "Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos". Biochemical and Biophysical Research Communications. 358 (2): 521–7. doi:10.1016/j.bbrc.2007.04.172. PMID 17493584.

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Antisense therapy

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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...

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Oligonucleotide

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properties to the oligonucleotides and make them a key element in antisense therapy. Oligonucleotides are chemically synthesized using building blocks...

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Cystic fibrosis

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function would provide an alternative to traditional antibiotic therapies. Antisense therapy is being researched to treat a subset of mutations which have...

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Antisense RNA

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Antisense RNA (asRNA), also referred to as antisense transcript, natural antisense transcript (NAT) or antisense oligonucleotide, is a single stranded...

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Pelacarsen

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Pelacarsen is an antisense therapy that is designed to reduce Lipoprotein(a) concentrations in people with high levels of the lipoprotein and who are...

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Ionis Pharmaceuticals

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head of research of GlaxoSmithKline, with a goal to commercialize antisense therapy. In 1992, the company received its first approval by the Food and...

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RNA interference

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known as precise, efficient, stable and better than antisense therapy for gene suppression. Antisense RNA produced intracellularly by an expression vector...

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Gene therapy

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procedural practices. The modified gene therapy strategy of antisense IGF-I RNA (NIH n˚ 1602) using antisense / triple helix anti-IGF-I approach was registered...

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Olpasiran

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Olpasiran (AMG890) is an experimental antisense therapy designed to lower the level of lipoprotein(a), which is believed to be a causal factor in the...

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Organothiophosphate

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phosphate moiety is replaced by sulfur. These compounds are the basis of antisense therapy, e.g., the drugs fomivirsen (Vitravene), Oblimersen, Alicaforsen,...

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Duchenne muscular dystrophy

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address the root cause are currently available including gene therapy (Elevidys), and antisense drugs (Ataluren, Eteplirsen etc.). Other medications used...

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Gapmer

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reactions, nausea, headache, fatigue, and fever Antisense therapy Oligonucleotide synthesis Antisense RNA therapeutics Crooke, Stanley T.; Baker, Brenda...

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Eteplirsen

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be used to treat about 14% of DMD cases. Eteplirsen is a form of antisense therapy. Eteplirsen was developed by Steve Wilton, Sue Fletcher and colleagues...

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Index of genetics articles

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strand Anticodon Antigen Antimorph Antiparallel Antisense Antisense RNA Antisense strand Antisense therapy AP endonuclease AP site Apert syndrome Apoptosis...

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Telomerase reverse transcriptase

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molecular genetic-based methods of inhibiting telomerase, such as antisense therapy and RNA interference. hTERT peptide fragments have been shown to induce...

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MECP2 duplication syndrome

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reversed MECP2 Duplication Syndrome in adult symptomatic mice using antisense therapy. Mice treated with an experimental ASO administered through the central...

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Antiviral drug

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other antisense antivirals are in development. An antisense structural type that has proven especially valuable in research is morpholino antisense. Morpholino...

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Exon skipping

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exons to be skipped could be used to treat up to 50% of DMD patients. Antisense therapy Wahl M (1 October 2011). "Exon Skipping in DMD: What Is It and Whom...

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Alexander disease

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gene editing of the astrocytes. A phase III clinical trial of an antisense therapy, sponsored by Ionis Pharmaceuticals, began in 2021. A bone marrow...

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Thiophosphate

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the phosphate moiety is replaced by sulfur. They are the basis of antisense therapy, e.g., the drugs fomivirsen (Vitravene), oblimersen, alicaforsen,...

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Gilead Sciences

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to target specific genetic code sequences – that is, antisense therapy, a form of gene therapy. According to Riordan, he had always wanted to use the...

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Antibiotic

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as immunoglobulin therapy, and experimental biology-based approaches such as phage therapy, fecal microbiota transplants, antisense RNA-based treatments...

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Growth hormone therapy

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Growth hormone therapy refers to the use of growth hormone (GH) as a prescription medication—it is one form of hormone therapy. Growth hormone is a peptide...

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Peptide nucleic acid

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recent years in molecular biology procedures, diagnostic assays, and antisense therapies. Due to their higher binding strength, it is not necessary to design...

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Index of topics related to life extension

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disease Antagonistic Pleiotropy Antioxidant Polyphenol antioxidant Antisense therapy Apoptosis Atherosclerosis ATP (adenosine triphosphate) Autoimmune...

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Gene silencing

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2007). "A review of antisense therapeutic interventions for molecular biological targets in asthma". Biologics: Targets and Therapy. 1 (3): 271–83. PMC 2721314...

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