Base editing therapies enter clinical trials for PCSK9 silencing to lower LDL-cholesterol
Updated
Updated · Nature.com · Apr 27
Base editing therapies enter clinical trials for PCSK9 silencing to lower LDL-cholesterol
6 articles · Updated · Nature.com · Apr 27
Early clinical trial results indicate these therapies may offer a durable, single-treatment solution for reducing plasma LDL-cholesterol in humans.
The trials target PCSK9, a gene regulating LDL receptor degradation, and initial data suggest significant and lasting cholesterol reduction.
This approach could transform treatment for hypercholesterolemia, building on prior successes in animal models and representing a major advance beyond current PCSK9 inhibitors and RNA-based therapies.
With a new oral pill also slashing cholesterol, is an irreversible gene therapy worth the gamble?
If we can edit genes to prevent heart attacks, which common chronic disease is next?
How can the FDA approve a permanent therapy when its true safety profile won't be known for 15 years?
This one-shot cure could save millions of lives, but will anyone actually be able to afford it?
One-time cures could upend the drug industry. Is our healthcare system ready for the financial shock?