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Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce SOD1 protein synthesis. Tofersen can be used in amyotrophic lateral sclerosis (ALS) research.
| Molecular Weight | 7128.00 |
| CAS Number | 2088232-70-4 |
| Solubility (25°C) | Water ≥ 60 mg/mL |
| Storage | -20°C, dry, sealed |
[1] Timothy M Miller, et al. N Engl J Med. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
[2] Johannes Dorst, et al. Curr Opin Neurol. Clinical studies in amyotrophic lateral sclerosis
[4] Daisuke Ito. Ann Neurol. Promise of Nucleic Acid Therapeutics for Amyotrophic Lateral Sclerosis
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