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CAS 1258984-36-9

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Nusinersen

Description:
Nusinersen is an antisense oligonucleotide used primarily in the treatment of spinal muscular atrophy (SMA), a genetic disorder characterized by the degeneration of motor neurons. It is designed to modify the splicing of the SMN2 gene, thereby increasing the production of the survival motor neuron (SMN) protein, which is crucial for the health of motor neurons. Nusinersen is administered via intrathecal injection, allowing it to bypass the blood-brain barrier and directly target the central nervous system. The chemical structure of Nusinersen consists of a modified RNA sequence, which enhances its stability and binding affinity to the target mRNA. Its mechanism of action involves hybridization to the SMN2 pre-mRNA, promoting the inclusion of exon 7 and leading to the production of a functional SMN protein. Nusinersen has been shown to improve motor function and survival in patients with SMA, making it a significant advancement in the treatment of this condition. Its development represents a novel approach in gene therapy, utilizing the principles of molecular biology to address genetic disorders.
Formula:C234H340N61O128P17S17
Synonyms:
  • NU
  • Nusinersen USP/EP/BP
  • Nusinersen
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Found 3 products.
  • Nusinersen

    CAS:
    <p>Nusinersen (nusinersen) is a antisense oligonucleotide (ASO) for the treatment of pediatric and adult spinal muscular atrophy (SMA) and increases SMN proteinss.</p>
    Purity:98.62%
    Color and Shape:Solid
  • Nusinersen sodium

    CAS:
    <p>An antisense oligonucleotide in which the 2'-positions in the ribose ring are protected with 2'-O-2-methoxyethyl groups and the phosphate linkages replaced with phosphorothioate linkages.  Used to treat spinal muscular atrophy.</p>
    Formula:C234H340N61O128P17S17•Na18
    Purity:Min. 95%
    Color and Shape:Powder
    Molecular weight:7,540.85 g/mol

    Ref: 3D-FN184231

    1mg
    191.00€
    5mg
    358.00€
    10mg
    509.00€
    25mg
    964.00€
    50mg
    1,420.00€
  • Nusinersen

    CAS:
    <p>An antisense oligonucleotide in which the 2'-positions in the ribose ring are protected with 2'-O-2-methoxyethyl groups and the phosphate linkages replaced with phosphorothioate linkages.  Used to treat spinal muscular atrophy.</p>
    Formula:C234H340N61O128P17S17
    Purity:Min. 95%
    Color and Shape:Powder
    Molecular weight:7,127.21 g/mol

    Ref: 3D-FN170919

    25mg
    3,169.00€