One-time CRISPR hit lowers cholesterol in monkeys

^{“Cholesterol emboli” by Boonyarit Cheunsuchon
is licensed under CC BY-NC 2.0}

A single shot of gene therapy lowers “bad” cholesterol by about 60% for at least 8 months in monkeys, a new study reports (Nature 2021, DOI: 10.1038/s41586-021-03534-y). The shot knocks down a gene called PCSK9, which stands for proprotein convertase subtilisin/kexin type 9. PCSK9 proteins typically bind receptors that break down artery-clogging low-density lipoprotein (LDL) cholesterol. Without functioning PCSK9, cells have more LDL receptors available to grab and remove LDL from the blood.

Currently, doctors treat high cholesterol with statins as well as antibodies to the PCSK9 protein, which people must take daily or weekly. A long-term treatment would improve patient adherence to therapies, the researchers write. The gene therapy would be particularly relevant for people with a rare condition called familial hypercholesterolaemia, which leads to consistently high cholesterol levels and is caused by mutations in PCSK9.

“This is a landmark study, with important implications for a one-time treatment for patients suffering from elevated cholesterol due to genetic reasons,” David R. Liu, chemical biologist...