For research use only. Not for therapeutic Use.
Tofersen（Cat No.:I045375）is an antisense oligonucleotide (ASO) therapeutic designed to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. By binding to SOD1 mRNA, Tofersen reduces the production of the toxic SOD1 protein, which is implicated in the progression of ALS. Administered via intrathecal injection, Tofersen targets the underlying genetic cause of SOD1-related ALS, offering a potential disease-modifying approach. Clinical trials have shown its ability to lower SOD1 levels, providing hope for improved outcomes in this devastating neurodegenerative disorder.
| Catalog Number | I045375 |
| CAS Number | 2088232-70-4 |
| Purity | 95% |
| Target | SOD1 |
| Target Protein | P00441,,, |
| Appearance | Solid |
| Storage | Dry, dark and at 2 - 8 °C for six months or -20°C for two years. |
| Reference | [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. |
