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.