Biotechnology & Genomics: Clinical trial results published in Nature Medicine show that targeted epigenetic methylation delivered via lipid nanoparticles successfully silenced the liver PCSK9 gene without introducing double-stranded DNA breaks.
Zero Double-Stranded DNA Cleavage
Rather than cutting chromosomes with Cas9 nucleases, the epigenetic editor fuses a dead-Cas platform with DNA methyltransferases, depositing epigenetic ‘off’ tags directly onto the gene promoter region.
Long-Term Cardiovascular Protection
Patients receiving a single intravenous infusion experienced an average 68% reduction in circulating LDL cholesterol, with durable suppression persisting through 24 months of follow-up.
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