"Long-lasting Gene Silencing and Cholesterol Control Through One-Time Epigenetic Editing in Mice"

Researchers have developed a hit-and-run epigenome editing strategy for durable and efficient gene silencing in vivo, using ZFP-based engineered transcriptional repressors (ETRs) to target the Pcsk9 gene in mice. The ZFP-based ETRs induced stable and specific epi-silencing of Pcsk9, resulting in reduced circulating levels of PCSK9 and LDL-associated cholesterol. The study also demonstrated the durability of epigenetic silencing even after active cell replication, paving the way for potential clinical applications of the epi-silencing technology.