Nusinersen (sodium)

Nusinersen sodium is an antisense oligonucleotide active molecule. Nusinersen sodium modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen sodium improves spinal muscular atrophy[1][2][3].

Product Specifications

UNSPSC

12352208

Target

DNA/RNA Synthesis

Type

Oligonucleotides

Related Pathways

Cell Cycle/DNA Damage

Applications

COVID-19-anti-virus

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/nusinersen-sodium.html

Purity

99.10

Solubility

H2O : ≥ 10 mg/mL

Smiles

[Nusinersen(sodium)]

Molecular Formula

C234H322N61Na18O128P17S17

Molecular Weight

7127.3 (free acid)

References & Citations

[1]Richard S Finkel, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377 (18) :1723-1732.|[2]Laura Torres-Benito, et al. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Am J Hum Genet. 2019 Jul 3;105 (1) :221-230.|[3]Berciano MT, et al. Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly (A) RNA distribution in a SMA mouse model. Sci Rep. 2020 Jul 1;10 (1) :10738.