“The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got brighter. In an initial trial, a single dose of a CRISPR gene-editing therapy lowered cholesterol levels without any serious side effects.

The ‘Heart-2’ trial – organised by the biotech company Verve Therapeutics in Boston – involved 14 people who either had an inherited condition leading to very high cholesterol or had developed heart disease at a young age. While this was an early-stage trial that is mainly designed to test safety, the results indicate a clear dose-dependent effect.

The treatment, called Verve-102, involves a form of CRISPR gene editing called base editing. It is designed to alter a single DNA letter in the genomes of cells in the liver, switching off the production of a protein called PCSK9.

In the four people given the lowest dose, levels of harmful low-density lipoprotein cholesterol (LDL-C) fell by an average of 21 per cent. For the six given a medium dose, LDL-C levels fell by 41 per cent, and for the four given the highest dose, they fell by 53 per cent.”

From New Scientist.