Researchers at the Cleveland Clinic have shared promising findings from the initial phase of a clinical trial involving CTX310, an innovative gene-editing therapy aimed at reducing cholesterol levels. The treatment, administered once via intravenous infusion, modifies the DNA within liver cells. Utilizing the CRISPR-Cas9 technique, the therapy effectively deactivates the ANGPTL3 gene, responsible for producing a protein that manages lipid levels in the bloodstream. Consequently, patients experience sustained lower levels of cholesterol and triglycerides without the need for ongoing treatments. A year after receiving the highest dosage, data from four participants revealed that their LDL, often referred to as "bad" cholesterol," remained approximately 52.5% lower than initial levels, while triglycerides dropped by 47.8%. Currently, the study has included 15 patients with lipid disorders that proved challenging to manage with standard therapies. Throughout the first year, researchers noted no significant adverse effects; however, participants will continue to be monitored for the next 15 years due to the permanence of the DNA alteration and the necessity to assess its long-term safety.
Informational material. 18+.