What is the CRISPR gene-editing therapy and how does it work?

The CRISPR gene-editing therapy targets a gene in the liver called ANGPTL3 to lower LDL "bad" cholesterol and triglycerides, both of which are linked to heart disease.

How effective was the CRISPR therapy in the clinical trial?

The CRISPR therapy was able to lower cholesterol and triglyceride levels by around 50% in the 15 participants, and the results were sustained for at least 60 days after a single dose.

Who were the participants in the CRISPR therapy trial?

The 15 participants were from Australia, New Zealand, and the UK, and they were all in their 50s and 60s with uncontrolled high cholesterol despite taking conventional medications.

Home / Health / Cutting-Edge Gene Therapy Slashes Cholesterol by 50% with Single Dose

Cutting-Edge Gene Therapy Slashes Cholesterol by 50% with Single Dose

11 Nov

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Summary

Experimental gene-editing treatment lowers LDL "bad" cholesterol and triglycerides by around 50%
Results sustained for at least 60 days after a single dose
Therapy could be "life-changing" for patients with uncontrolled high cholesterol

Cutting-Edge Gene Therapy Slashes Cholesterol by 50% with Single Dose

A cutting-edge gene-editing treatment has demonstrated the ability to dramatically slash harmful cholesterol levels by around 50% in a small clinical trial. The experimental therapy, which uses the CRISPR gene-editing tool to target a key gene in the liver, has provided significant reductions in LDL "bad" cholesterol and triglycerides for at least 60 days after a single dose.

The trial involved 15 participants from Australia, New Zealand, and the UK, all in their 50s and 60s, who had uncontrolled high cholesterol despite taking conventional medications. Researchers reported that the participants experienced a rapid drop in their cholesterol and triglyceride levels within just two weeks of receiving the treatment, with the effects sustained for at least two months.

According to the lead researcher, Dr. Steven Nissen of the Cleveland Clinic, this type of therapy could be "life-changing" for many patients struggling to manage their high cholesterol. Unlike daily pills or monthly injections, this gene-editing treatment offers a potential "one-and-done" solution that is both safe and durable. While the trial was small, the scientists are optimistic that future successful studies could pave the way for a revolutionary new approach to cholesterol management.

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