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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]
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]
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 ...
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.
Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [2] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis.
The approval of aducanumab, despite the lack of evidence to support its therapeutic effects, led to controversy about the FDA’s approval process and a reluctance to prescribe the drug.
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 ...