| Bioactivity | Tominersen (RG6042) is a second-generation 2′-O-(2-methoxyethyl) antisense oligonucleotide that targets huntingtin protein (HTT) mRNA and potently suppresses HTT production. Tominersen improves survival and reduces brain atrophy in mice. Tominersen can be used for the research of Huntington’s disease (HD)[1][2]. |
| Invitro | Tominersen binds to its cognate mRNA by means of Watson-Crick base-pair interactions, triggering RNase H1-mediated degradation of the target mRNA[1]. |
| In Vivo | Tominersen can be detected in the neurons of most brain regions, including the frontal cortex, striatum, thalamus, midbrain, brainstem and cerebellum after delivering into the CNS[2].Tominersen effectively suppresses huntingtin accumulation, and generates sustained phenotypic reversal in HD-like disease after transient huntingtin reduction in rodents[2]. |
| Name | Tominersen |
| CAS | 1709886-74-7 |
| Molar Mass | 7078.00 |
| Appearance | Solid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | -20°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
| Reference | [1]. Tabrizi SJ, et, al. Targeting Huntingtin Expression in Patients with Huntington's Disease. N Engl J Med. 2019 Jun 13;380(24):2307-2316. [2]. Kordasiewicz HB, et al. Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron. 2012 Jun 21;74(6):1031-44. |