Tofersen (Synonyms: BIIB067; ISIS-SOD1Rx) |
| Katalog-Nr.GC64897 |
Tofersen (BIIB067) ist eine Antisense-Oligonukleotid, das den RNase H-abhängigen Abbau von Superoxiddismutase 1 (SOD1)-mRNA vermittelt, um die Synthese des SOD1-Proteins zu reduzieren.
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Cas No.: 2088232-70-4
Sample solution is provided at 25 µL, 10mM.
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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].
Tofersen targets the SOD1 mRNA using antisense oligonucleotides (ASOs) that bind to the SOD1 mRNA by Watson-Crick base pairing. Tofersen activates RNase H1 that destroys the targeted RNA[2].
[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.
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