Oligonucleotide Therapeutics (ASO/siRNA)

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.

2 Items

  • Nusinersen
    Cat. No.
    A37665

    Nusinersen

    Nusinersen is an antisense oligonucleotide that targets the SMN2 gene to enhance pre-messenger RNA splicing. By promoting the production of full-length survival motor neuron (SMN) protein, Nusinersen plays a crucial role in the treatment of spinal muscular atrophy (SMA). This compound is valuable in research focused on genetic disorders related to neuronal degeneration and SMN protein deficiency.

    1 mg
    $250.00
    5 mg
    $645.00
    All 5 sizes
  • Remlarsen
    Cat. No.
    A57838

    Remlarsen

    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.

    1 mg
    $95.00
    5 mg
    $260.00
    All 3 sizes

2 Items

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