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). In Vitro Tominersen binds to its cognate mRNA by means of Watson-Crick base-pair interactions, triggering RNase H1-mediated degradation of the target mRNA. MCE has not independently confirmed the accuracy of these methods. They are for reference only. 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. Tominersen effectively suppresses huntingtin accumulation, and generates sustained phenotypic reversal in HD-like disease after transient huntingtin reduction in rodents. MCE has not independently confirmed the accuracy of these methods. They are for reference only. Form:Solid