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DNA,d(rGm-[P(S)]-sp-rGm-rCm-rAm-rCm-[P(S)]-sp-A-[P(S)]-sp-A-[P(S)]-sp-G-[P(S)]-sp-G-[P(S)]-sp-G-[P(S)]-sp-C-[P(S)]-sp-A-[P(S)]-sp-C-[P(R)]-sp-A-[P(S)]-sp-G-[P(S)]-sp-rAm-rCm-rUm-rUm-[P(S)]-sp-rCm) |
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Rovanersen (WVE-120101) is an antisense oligonucleotide that specifically targets mutated mRNA copies of the huntington (HTT) gene without affecting healthy mRNA of HTT gene, thereby preventing the production of faulty Huntingtin protein. Rovanersen can be used for huntington’s disease research[1]. | [in vivo]
In non-human primates (NHPs), Rovanersen (WVE-120101) is found in nuclear and perinuclear compartments of neurons in brain areas believed to be involved in HD pathology[1]. | [References]
[1] M Meena, et al. Selectivity and Biodistribution of WVE-120101, a Potential Antisense Oligonucleotide Therapy for the Treatment of Huntington’s Disease (S56.001). AAN Enterprises. April 18, 2017; 88 (16 Supplement). |
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