Design of optimized epigenetic regulators for durable gene silencing with application to PCSK9 in nonhuman primates.
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Optimized TALE-based epigenetic regulators achieve 98% gene silencing with durable effects over 343 days in primates.
- Why it matters: Effective, long-lasting gene silencing without permanent DNA changes addresses safety concerns of genome editing for treating human diseases.
- What they did: Researchers designed and tested epigenetic regulators combining TALE and dCas9 effectors, demonstrating high efficiency in mice and macaques, with minimal off-target effects.
- The result: A single dose of TALE-based EpiReg silenced PCSK9 in primate liver for over a year, paving the way for epigenetic therapies adaptable to various genes and conditions.