Nusinersen

Modify Date: 2025-08-20 12:36:07

Nusinersen Structure
Nusinersen structure
Common Name Nusinersen
CAS Number 1258984-36-9 Molecular Weight 7127.194237
Density N/A Boiling Point N/A
Molecular Formula C234H340N61O128P17S17 Melting Point N/A
MSDS N/A Flash Point N/A

 Use of Nusinersen


Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein[1].

 Names

Name Nusinersen
Synonym More Synonyms

 Nusinersen Biological Activity

Description Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein[1].
Related Catalog
In Vitro Nusinersen is an SMN-ASO that increases SMN levels by blocking an intronic splice silencer in SMN2, thereby facilitating the exon 7 inclusion and generation of FL-SMN2 transcripts[2].
References

[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.

 Chemical & Physical Properties

Molecular Formula C234H340N61O128P17S17
Molecular Weight 7127.194237
Storage condition -20°C

 Synonyms

Nusinersen
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