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Oligonucleotide Therapeutics lists antisense oligonucleotides and small interfering RNAs that reduce or modify gene expression. Each antisense oligonucleotide (ASO) binds complementary RNA and, depending on its chemistry, triggers ribonuclease H (RNase H) cleavage, alters splicing or blocks translation. Antisense examples include eteplirsen, a phosphorodiamidate morpholino oligomer, and danvatirsen, which is directed at signal transducer and activator of transcription 3 (STAT3). Each small interfering RNA (siRNA) is a short double-stranded RNA that guides the RNA-induced silencing complex (RISC) to cleave a matching messenger RNA (mRNA). Inclisiran, for example, is an siRNA directed at proprotein convertase subtilisin/kexin type 9 (PCSK9). Phosphoramidite building blocks for oligonucleotide synthesis are listed separately under Phosphoramidites.
Synonyms: MRG-201
Remlarsen (MRG-201) is a miR-29b mimic that functions as an agonist of the miR-29b pathway. It is primarily involved in inhibiting the formation of fibrotic scars and addressing cutaneous fibrosis. This reagent is valuable for research applications focused on fibrosis-related conditions and the modulation of miRNA pathways in cellular processes.
Synonyms: STK-001 sodium
Zorevunersen sodium is an antisense oligonucleotide that acts as an activator of the Scn1a gene through TANGO technology. This compound enhances Scn1a mRNA transcripts, leading to increased expression of the NaV1.1 protein. By restoring the excitability of parvalbumin interneurons, Zorevunersen sodium effectively reduces seizure frequency and improves survival in mouse models. It is a valuable tool for research focused on Dravet syndrome and related epileptic disorders.
Synonyms: STK-001
Zorevunersen is an antisense oligonucleotide that acts as an activator of the Scn1a gene through TANGO technology. It enhances the production of Scn1a mRNA transcripts, leading to an increased expression of the NaV1.1 protein. This compound has demonstrated the ability to restore the excitability of parvalbumin interneurons, effectively reducing seizure activity and improving survival rates in murine models. Zorevunersen is a valuable tool for research focused on Dravet syndrome and related channelopathies.
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