Votoplam (PTC518) is a blood-brain barrier-permeable HTT pre-mRNA inhibitor with an IC 50 ≤ 0.1 μM. Votoplam modulates the splicing process of HTT pre-mRNA to include a pseudo-exon carrying a premature translation termination codon, thereby triggering HTT mRNA degradation. Votoplam is applicable to research related to Huntington's disease.
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