Do you have high cholesterol? With gene editing, there soon may be a CRISPR cure

Credit: Charlotte Pollet for Bloomberg Businessweek
Credit: Charlotte Pollet for Bloomberg Businessweek

A trial testing a new CRISPR-based treatment to lower cholesterol has officially kicked off in New Zealand. If it works as well as it did in animal trials, the one-and-done treatment could save countless lives — permanently lowering cholesterol and the risk of a heart attack.

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Verve Therapeutics’ cholesterol treatment… is designed to permanently deactivate a gene in the liver that controls the production of PCSK9 — a protein that prevents the removal of excess cholesterol from the body.

In monkey trials, it reduced LDL cholesterol levels by 70% in just two weeks and kept them low for at least two years — suggesting that it could effectively cure high cholesterol in people.

The delivery mechanism for Verve’s treatment to lower cholesterol is also notable. 

Instead of taking the traditional approach to gene therapy, using a hollowed-out virus to deliver its CRISPR therapy, the company uses nanoparticles to deliver genetic instructions into cells, just like the ones used to package the mRNA vaccines for COVID-19.

The big picture: Therapies that work in animals often don’t translate to humans, and the permanence of Verve’s treatment means there is no undo button.

This is an excerpt. Read the original post here

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