CRISPR Therapy Lowers Cholesterol: Trial Results

by Archynetys Health Desk
Illustration (Doc Kemkes.go.id)

High cholesterol is one of the main risk factors for heart disease and stroke. Cholesterol levels can be controlled with a healthy diet and lifestyle changes.

One of the main approaches is through a cholesterol diet, namely adjusting your diet to reduce levels of bad cholesterol (Low-Density Lipoprotein/LDL). It is called bad cholesterol, because it can clog blood vessels.

In the National Library of Medicine (NIH) journal entitled Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 which involved 15 patients with high cholesterol conditions who were given the CRISPR-Cas9 drug which is said to be able to cut genes.

Quoted from the American College of Cardiology, the drug is given via a one-time intravenous fluid therapy that targets angiopoietin-like protein 3 (ANGPTL3) is safe and reduces LDL cholesterol by almost 50% and reduces triglycerides by around 55%.

The phase 1 trial was the first in humans, involving 15 participants with uncontrolled hypercholesterolemia, hypertriglyceridemia, or dyslipidemia who received maximally tolerated lipid-lowering therapy. Each participant received one intravenous dose of CTX310 with a mean follow-up of at least 60 days.

In presenting the results, the researchers stated that the primary endpoint of serious adverse events occurred in two participants, while there were no dose-limiting toxic effects associated with CTX310.

Reductions in LDL cholesterol and triglycerides occurred at all dose levels, with initial reductions appearing within the first two weeks of treatment and persisting for at least 60 days.

“Adherence to cholesterol-lowering therapy is one of the biggest challenges in preventing heart disease,” said one of the researchers, Steven E. Nissen, quoted on Thursday (13/11).

“Many patients stop taking their cholesterol medication within the first year. The possibility of a one-time treatment with long-term effects could be a significant clinical improvement,” he continued.

Meanwhile, Stephen J. Nicholls called this finding unprecedented and could change treatment for people suffering from LDL cholesterol.

“If confirmed in a larger trial, this approach could change care for people with lifelong lipid disorders and drastically reduce cardiovascular risk,” he concluded. (D-2)

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