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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 |
| 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. |
Dissolve 2 mg of the compound in 100 μL DMSO
to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
1) Add 100 μL of the DMSO
stock solution to 400 μL PEG300
and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O
and mix thoroughly until a homogeneous solution is obtained.
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