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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]
Nusinersen is an antisense oligonucleotide in which the 2'-hydroxy groups of the ribofuranosyl rings ... [22] It was approved by the FDA in December 2016 and by EMA ...
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 ...
Tegsedi, developed and marketed by Ionis Pharmaceuticals, was approved by the FDA in October 2018 for the treatment of hereditary transthyretin amyloidosis (hATTR). [10] The chemical structure is a 20-mer oligonucleotide with PS backbone modifications and 2'-MOE ribose substitutions. [4]
Aganirsen is a 25-mer (5'-TATCCGGAGGGCTCGCCATGCTGCT-3'), first-generation antisense oligonucleotide (phosphorothioate linkage). Aganirsen has a molecular mass of 8036 (Da), a melting point of approximately 64.2 °C, and a GC content of 64%. Aganirsen is highly soluble in water.
Viltolarsen was approved for medical use in the United States in August 2020. [2] [3] After golodirsen was approved in December 2019, viltolarsen is the second approved targeted treatment for people with this type of mutation in the United States. [2] [4] Approximately 8% of people with DMD have a mutation that is amenable to exon 53 skipping. [2]
New Drug Applications (NDA) for eteplirsen and a similar drug drisapersen were filed with the US Food and Drug Administration (FDA) in August 2015. [13] The Prescription Drug User Fee Act (PDUFA) goal dates for these were December 27, 2015 for drisapersen and February 26, 2016, for eteplirsen. Following FDA rejection of drisapersen, the agency ...
The antisense oligonucleotide drug oblimersen was developed by Genta Incorporated to target Bcl-2. An antisense DNA or RNA strand is non-coding and complementary to the coding strand (which is the template for producing respectively RNA or protein).
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related to: fda approved antisense oligonucleotides