1258984-36-9

1258984-36-9 structure

Name: Nusinersen
Chemical Name: Nusinersen
CAS Number: 1258984-36-9
Molecular Formula: C₂₃₄H₃₄₀N₆₁O₁₂₈P₁₇S₁₇
Molecular Weight: 7127.194237
Catalog: API Nervous system medication Other nervous system medication
Create Date: 2018-06-08 15:08:06
Modify Date: 2025-08-20 12:36:07
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].

Name	Nusinersen
Synonyms	Nusinersen

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	Signaling Pathways >> Cell Cycle/DNA Damage >> DNA/RNA Synthesis Research Areas >> Inflammation/Immunology
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.