Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

Genome editing in non-human primates with a PCSK9-targeted meganuclease lowers cholesterol levels. Clinical translation of in vivo genome editing to treat human genetic diseases requires thorough preclinical studies in relevant animal models to assess safety and efficacy. A promising approach to treat hypercholesterolemia is inactivating the secreted protein PCSK9, an antagonist of the LDL receptor. Here we show that single infusions in six...