Experimental One-Dose Treatment Cuts ‘Bad’ Cholesterol by 52% in First Human Trial
This could be a breakthrough.
“`html
Researchers have demonstrated promising results from an experimental one-dose genetic treatment designed to lower harmful cholesterol levels. The therapy, called CTX310, uses CRISPR gene-editing technology to make permanent modifications that mimic a naturally occurring genetic variant found in some individuals. This variant reduces production of a liver protein called ANGPTL3, enabling the body to process blood fats more efficiently.
In a clinical trial led by the Cleveland Clinic Coordinating Center for Clinical Research, 15 patients with serious cholesterol and triglyceride disorders received an intravenous infusion of nanoparticles containing CRISPR instructions. Among the four participants who received the highest dose, LDL cholesterol—commonly known as “bad” cholesterol—decreased by an average of 52.5 percent. Triglyceride levels also improved, falling by approximately 47.8 percent. The results remained stable one year after treatment.
The findings, published in the New England Journal of Medicine, suggest CTX310 could offer a significant advantage over existing treatments, which typically require daily or weekly administration. One-time treatment could simplify patient compliance for those whose conditions have not responded to conventional medications. Researchers noted that few adverse events occurred during the study period, though they acknowledge that larger trials involving more participants over extended periods are necessary before the therapy can be considered for wider use.
A second phase of testing is currently underway to further evaluate the treatment’s safety and effectiveness in additional patient populations with specific lipid disorders.
“`
Originally reported by Sciencealert →
About the Author
