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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 most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain. [ 2 ] [ 5 ] Casimersen was approved for medical use in the United States in February 2021, [ 1 ] [ 2 ] [ 6 ] and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene ...
Gapmer-based therapeutics also have the potential for side effects. For example, Kynamro has been shown to induce injection site reactions, nausea, headaches, flu-like symptoms, and hepatotoxic reactions. [9] Side effects of Inotersen include thrombocytopenia, glomerulonephritis, injection site reactions, nausea, headache, fatigue, and fever [10]
An example of an RNA aptamer therapy includes Pegaptanib (aka Macugen ® ), the only FDA-approved RNA aptamer treatment. [7] [8] [87] Originally approved in 2004 to treat age-related macular degeneration, Pegaptanib is a 28 nucleotide RNA aptamer that acts as a VEGF antagonist.
[1] [2] Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide. [1] It is given by injection under the skin. [1] Olezarsen was approved for medical use in the United States in December 2024. [1] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4]
The US Food and Drug Administration (FDA) approved inotersen in October 2018. [6] The application for inotersen was granted orphan drug designation. [10] The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 participants with hereditary transthyretin-mediated amyloidosis. [6]
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 ...
Golodirsen is one of the very few FDA-approved exon-skipping therapy for Duchenne muscular dystrophy, although the clinical benefits of the medication are yet to established. [ 1 ] [ 3 ] While the development of golodirsen needed huge financing, it is only applicable to a small subset of people with Duchenne muscular dystrophy.