| Chemical Properties | Back Directory | [storage temp. ]
-20°C, stored under nitrogen | [form ]
Solid | [color ]
Off-white to light yellow | [Water Solubility ]
Water : 100 mg/mL (14.03 mM; Need ultrasonic) |
| Hazard Information | Back Directory | [Uses]
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]. | [References]
[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. DOI:10.1056/NEJMoa2003715
[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. DOI:10.1172/JCI99081 |
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