Experimental Gene Therapy Reduces Cholesterol and Triglyceride Levels - Sarmad

A study conducted by physicians and researchers at the U.S.-based Cleveland Clinic, in collaboration with researchers from Australia and New Zealand, has shown that a single dose of an experimental gene therapy contributed to lowering levels of harmful cholesterol and triglycerides in individuals with lipid disorders.

According to MedicalXpress, the study included 15 participants in a Phase I clinical trial who received a single dose of the experimental therapy, CTX310, to assess its safety and impact on blood lipid levels.

The treatment relies on the CRISPR-Cas9 gene-editing technology, which allows for precise modifications to DNA. It targets a gene called ANGPTL3 in liver cells, which is involved in regulating blood lipid levels.

Twelve months after receiving the treatment, participants who received the highest dose showed a significant reduction in low-density lipoprotein (LDL) cholesterol, commonly known as “bad” cholesterol, as well as in triglyceride levels—a type of fat found in the blood—compared to baseline measurements taken before treatment.

Additionally, levels of the ANGPTL3 protein, which plays a role in regulating blood lipids, decreased among participants who received the highest dose, indicating that the therapy’s effect on the targeted gene persisted throughout the follow-up period.

These findings suggest that gene-editing technologies could potentially be used in the future to lower blood lipid levels with a single dose. Research on the therapy is ongoing to further evaluate its safety and efficacy on a broader scale.