Shelly Fan in Singularity Hub:
CRISPR-based therapies just hit another milestone. In a small clinical trial with 10 people genetically prone to dangerously high levels of cholesterol, a single infusion of the precision gene editor slashed the artery-clogging fat by up to 55 percent. If all goes well, the one-shot treatment could last a lifetime.
The trial, led by Verve Therapeutics, is the first to explore CRISPR for a chronic disease that’s usually managed with decades of daily pills. It also marks the first use of a newer class of gene editors directly in humans. Called base editing, the technology is more precise—and potentially safer—than the original set of CRISPR tools. The new treatment, VERVE-101, uses a base editor to disable a gene encoding a liver protein that regulates cholesterol.
To be clear, these results are just a sneak peek into the trial, which was designed to test for safety, rather than the treatment’s efficacy. Not all participants responded well. Two people suffered severe heart issues, with one case potentially related to the treatment. Nevertheless, “it is a breakthrough to have shown in humans that in vivo [in the body] base editing works efficiently in the liver,” Dr. Gerald Schwank at the University of Zurich, who wasn’t involved in the trial, told Science.
More here.