
CAS 1258984-36-9
: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:SolidNusinersen 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•Na18Purity:Min. 95%Color and Shape:PowderMolecular weight:7,540.85 g/molNusinersen
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:C234H340N61O128P17S17Purity:Min. 95%Color and Shape:PowderMolecular weight:7,127.21 g/mol

