Tofersen
Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. Tofersen can be used for the research of amyotrophic lateral sclerosis (ALS) [1].
Product Specifications
CAS Number
2088232-70-4
Product Name Alternative
BIIB067; ISIS-SOD1Rx; ISIS 333611
UNSPSC
12352208
Target
DNA/RNA Synthesis
Type
Oligonucleotides
Related Pathways
Cell Cycle/DNA Damage
Applications
Neuroscience-Neurodegeneration
Field of Research
Neurological Disease
Assay Protocol
https://www.medchemexpress.com/tofersen.html
Purity
96.53
Solubility
H2O : 100 mg/mL (ultrasonic)
Smiles
[Tofersen]
Molecular Formula
C230H317N72O123P19S15
Molecular Weight
7128.00
References & Citations
[1]Miller T, et, al. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2020 Jul 9;383 (2) :109-119. |[2]McCampbell A, et, al. Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models. J Clin Invest. 2018 Aug 1;128 (8) :3558-3567.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, stored under nitrogen, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Launched
Available Sizes
Frequently Asked Questions
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