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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: ISIS 766720 sodium; IONIS-GHR-LRx sodium
Cimdelirsen sodium is an investigative antisense oligonucleotide designed to inhibit growth hormone receptor (GHR) synthesis through a ligand-conjugated, hepatic-targeted mechanism. This compound effectively reduces the detrimental effects associated with growth hormone (GH) hypersecretion and lowers circulating insulin-like growth factor-1 (IGF-1) levels in patients with acromegaly. Its primary applications include studying the modulation of GH signaling and exploring therapeutic strategies in disorders related to GH excess.
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