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编 号:F748676
分子量:6617
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CAS No: 2072901-32-5
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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.
体内研究:
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.
体外研究:
In vitro, Rovanersen (WVE-120101) leads to the selective RNase H cleavage of mutant Huntingtin allele (mHTT) over wtHTT, with no complement activation. Rovanersen selectively decreases mHTT mRNA and protein levels compared with wtHTT in multiple cells lines.