CRISPR base editors delivered via a single infusion of lipid nanoparticles to the liver efficiently reduced expression of the PCSK9 gene in monkeys. Researches observed a reduction of low-density lipoprotein cholesterol of approximately 60% that was stable after 8 months. Atherosclerotic cardiovascular disease is the leading cause of death worldwide.
Musunuru, K., Chadwick, A.C., Mizoguchi, T. et al. In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates. Nature 593, 429–434 (2021). https://doi.org/10.1038/s41586-021-03534-y
Keywords: CRISPR, Base Editors, Cholesterol
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