Research Highlights: CRISPR Editing Lowers LDL Cholesterol in Monkeys

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CRISPR Editing Lowers LDL Cholesterol in Monkeys

  • CRISPR is a technology that has changed the way basic research is conducted and the way we can now think about disease treatment.[2]
  • CRISPR can potentially modify genes that cause disease.
  • Durable gene editing in target organs of non-human primates is a key step before the administration of gene editor substances to humans.
  • Researchers demonstrated that CRISPR editors that are transported using lipid nanoparticles can modify genes that may cause diseases in living monkeys.
  • They observed that a gene called PCSK9 in the liver had reduced its expression activity after infusion of lipid nanoparticles.
  • PCSK9 is a gene that provides instructions for making a protein associated with the regulation of cholesterol in the blood.[3]
  • They also observed approximately 90% reduction of PCSK9 blood levels and about 60% reduction of low-density lipoprotein cholesterol.
  • All the changes observed remained stable for more than 8 months after a single-dose treatment.
  • The findings support a once-and-done approach to the reductions of LDL cholesterol and the atherosclerotic cardiovascular disease treatment.
  • Atherosclerotic cardiovascular disease is the leading cause of death worldwide.
  • The results also provide additional evidence for how CRISPR base editors can be applied to make single-nucleotide changes in therapeutic target genes in the liver, and potentially in other organs.

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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).