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Tofersen (BIIB067) is an antisense oligonucleotide (ASO) that mediates RNase H-dependent degradation of SOD1 (superoxide dismutase 1) mRNA, inhibiting SOD1 protein synthesis for potential use in amyotrophic lateral sclerosis (ALS).

| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 1 mg | $120 | - | In Stock | |
| 5 mg | $334 | - | In Stock | |
| 10 mg | $535 | - | In Stock | |
| 25 mg | $1,070 | - | In Stock | |
| 50 mg | $1,710 | - | In Stock | |
| 100 mg | $2,740 | - | In Stock |
| Description | Tofersen (BIIB067) is an antisense oligonucleotide (ASO) that mediates RNase H-dependent degradation of SOD1 (superoxide dismutase 1) mRNA, inhibiting SOD1 protein synthesis for potential use in amyotrophic lateral sclerosis (ALS). |
| In vitro | Tofersen targets SOD1 mRNA via antisense oligonucleotides (ASOs), binding through Watson-Crick base pairing. It activates ribonuclease H1 to degrade target RNA [2]. |
| Synonyms | ISIS-SOD1Rx, ISISSOD1Rx, ISIS333611, ISIS 333611, BIIB-067, BIIB067 |
| Cas No. | 2088232-70-4 |
| Color | White |
| Appearance | Solid |
| Sequence | DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2- methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]rA-sp-Tsp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU) |
| Storage | keep away from moisture | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
| Solubility Information | H2O: 80 mg/mL, Sonication is recommended. |
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