Cholesterol down – ready set edit!

Last week Verve Therapeutics dosed the first patient with a candidate treatment for hypercholesterolemia. This is exciting news for a couple of reasons. First, the technology used: CRISPR 2.0, i.e., base-editing is hitting the clinic (see the news in Nature Reviews Drug Discovery). Second, this is a leap forward into common diseases (“CRISPR for the masses”, says The Washington Post) and a training session for the real challenge, which is to “stop the biggest killer on Earth”, cardiovascular disease (MIT Technology Review).

CRISPR Express: nanovectors are coming

nanoparticle MIT[1423]Suppose you have developed the winning weapon to defeat certain genetic diseases by reliably correcting pathogenic mutations. There is still a problem: how do you march onto the battlefield, inside sick cells? The weapon is the genome-editing machinery, and the most efficient vessel ever tested are lipid nanoparticles. With this approach, described in a study published in Nature Biotechnology last week, CRISPR has beaten its success record in adult animals, knocking out the target gene in about 80% of liver cells. Continue reading