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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.

1 Item

  • Nusinersen
    Cat. No.
    A37665
    CAS No.
    1258984-36-9

    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.

    In stock View Details
    1 mg
    $250.00
    5 mg
    $645.00
    All 5 sizes

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