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Rovanersen is an antisense oligonucleotide designed to selectively target and degrade mutant mRNA transcripts of the huntingtin (HTT) gene, which is implicated in Huntington's disease. By binding specifically to the mutated HTT mRNA, rovanersen promotes its degradation via RNase H-mediated cleavage, thereby reducing the production of toxic mutant huntingtin protein while sparing wild-type HTT expression. Rovanersen is being investigated as a potential disease-modifying therapy for Huntington's disease and is currently in clinical development[2][4]. The drug was developed by Wave Life Sciences.
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