Home Epigenetics DNA/RNA Synthesis chemical Nusinersen

research use only

Nusinersen DNA/RNA Synthesis chemical

Cat.No.E6580

Nusinersen is an 18-mer, 2′-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide used for treating spinal muscular atrophy (SMA). It binds specifically to the intronic splice silencer N1 (ISS-N1) site in intron 7 of SMN2 pre-mRNA, blocking the binding of splicing repressors hnRNP A1/A2. This promotes inclusion of exon 7 during splicing, restoring full-length SMN protein production in SMA patients.
Nusinersen DNA/RNA Synthesis chemical Chemical Structure

Molecular Weight: 7124.42

Jump to

Quality Control

Molarity Calculator

Mass Concentration Volume Molecular Weight
Dilution Calculator Molecular Weight Calculator

In vivo
Batch:

In vivo Formulation Calculator (Clear solution)

Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)

mg/kg
g
μL

Step 2: Enter the in vivo formulation (This is only the calculator, not formulation. Please contact us first if there is no in vivo formulation at the solubility Section.)

% DMSO
%
% Tween 80
% ddH2O
% DMSO
+
%

Calculation results:

Working concentration: mg/ml;

Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.

Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such
as vortex, ultrasound or hot water bath can be used to aid dissolving.

Chemical Information, Storage & Stability

Molecular Weight 7124.42 Formula Storage (From the date of receipt)
CAS No. 1258984-36-9 -- Storage of Stock Solutions

Read more about storage stability stock solution CAS number

Mechanism of Action

References

Clinical Trial Information

(data from https://clinicaltrials.gov, updated on 2026-05-01)

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07047144 RECRUITING
Spinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type 3; Neuromuscular Manifestations; Anti-myostatin
Scholar Rock, Inc.
 2025-09-15 PHASE2
NCT05866419 RECRUITING
Spinal Muscular Atrophy; Spine Deformity; Scoliosis
Alcyone Therapeutics, Inc
 2023-11-27
NCT06555419 RECRUITING
Muscular Atrophy, Spinal
Biogen
 2025-01-16 PHASE1
NCT05067790 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy
Biogen
 2022-01-21 PHASE3
NCT04729907 COMPLETED
Muscular Atrophy, Spinal
Biogen
 2021-04-19 PHASE3
NCT05522361 UNKNOWN
Spinal Muscular Atrophy
Clinic for Special Children
 2022-11-06 PHASE4

Read more about Clinical Trials

Tech Support

Handling Instructions

Tel: +1-832-582-8158 Ext:3

If you have any other enquiries, please leave a message.

Signaling Pathway Map