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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Sources:
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
[2] https://sitn.hms.harvard.edu/flash/2014/crispr-a-game-changing-genetic-engineering-technique
