Researchers at the University of Pennsylvania, working with Verve Therapeutics, tested a single infusion of a gene-editing tool wrapped in a fat nanoparticle, designed to permanently disable the PCSK9 gene inside the liver cells of cynomolgus monkeys.
The edited monkeys showed the PCSK9 protein nearly disappear from their blood, down about 90 percent, and their LDL cholesterol, the harmful kind, fell by around 60 percent. The effect came from a single treatment and held steady for at least eight months of follow-up.
Unlike cholesterol pills, which only work while a person keeps taking them, a one-time DNA edit changes the gene itself, so the effect does not need to be renewed. Related treatments have since entered early trials in people with inherited high cholesterol; one version was paused in 2024 over lab abnormalities, and long-term safety in humans is still being established.
Read the original study
Musunuru et al., Nature, 2021 · doi.org/10.1038/s41586-021-03534-y
One shot, and the gene stayed off for months.
Source
- Musunuru, K., Chadwick, A. C., Mizoguchi, T., Garcia, S. P., DeNizio, J. E., Reiss, C. W., Wang, K., Iyer, S., Dutta, C., Clendaniel, V., Amaonye, M., Beach, A., Berth, K., Biswas, S., Braun, M. C., Chen, H.-M., Colace, T. V., Ganey, J. D., Gangopadhyay, S. A., . . . Kathiresan, S. (2021). In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates. Nature, 593(7859), 429–434. https://doi.org/10.1038/s41586-021-03534-y