Gene-Editing Technique Dramatically Lowers LDL Cholesterol Levels in Mice

If this works in humans, the implications are enormous.

CRISPR is a new technique for inactivating or editing specific genes. Developed in microorganisms, it also works in mammalian cells, including in vitro human cells (NEJM JW Gen Med Feb 6 2014) and monkey embryos (NEJM JW Gen Med Feb 13 2014).

About 10 years ago, a gene was identified that is critical to production of the LDL cholesterol receptor (LDL-R): PCSK9. Naturally occurring human mutations that enhance the effect of this gene greatly reduce production of LDL-R in the liver, causing hypercholesterolemia. Conversely, mutations that impair expression of the gene greatly increase production of LDL-R, leading to LDL cholesterol levels that are 40% lower than normal (and coronary heart disease rates that are 90% lower than normal).

A multi-i…

Reviewing Author

Anthony L. Komaroff, MD

Disclosures

Consultant/Advisory BoardSerImmune Inc.; DxTerity

Grant / Research SupportNIH (1U54NS105542-01)

Editorial BoardsHarvard Health Letter; UpToDate

Anthony L. Komaroff, MD

Disclosures

Consultant/Advisory BoardSerImmune Inc.; DxTerity

Grant / Research SupportNIH (1U54NS105542-01)

Editorial BoardsHarvard Health Letter; UpToDate

Gene-Editing Technique Dramatically Lowers LDL Cholesterol Levels in Mice

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Reviewing Author