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CRISPR for high cholesterol: 10 Breakthrough Technologies 2023

New forms of the gene-editing tool could enable treatments for common diseases.

Amrita Marino


Verve Therapeutics, Beam Therapeutics, Prime Medicine, Broad Institute


10 to 15 years

Last year, a New Zealand woman became the first to receive a gene-editing treatment to permanently lower her cholesterol. The woman had heart disease, along with an inherited risk for high cholesterol. But scientists behind the experimental treatment think it could help pretty much anyone.  

The trial is a potential turning point for CRISPR, the editing tool they used. Since the technology was first programmed to edit genomes about a decade ago, we’ve seen CRISPR move from scientific labs to clinics. But the first experimental treatments have focused on rare genetic disorders. A high-cholesterol treatment has wider potential. 

The cholesterol-lowering treatment, developed by Verve Therapeutics, relies on a form of gene editing called base editing, or “CRISPR 2.0.” It’s a more targeted approach—instead of simply making cuts to shut off specific genes, scientists can now swap a single DNA base for another. In theory, this should be safer because you’re less likely to cut an important gene by mistake, and you can avoid potential errors that may occur when DNA repairs itself after being cut. 

An even newer form of CRISPR could take things further still. Prime editing—or “CRISPR 3.0”—allows scientists to insert chunks of DNA into a genome. If it works in people, it could let scientists replace disease-causing genes.  

Together, these newer forms of CRISPR could broaden the scope of gene editing to take on many conditions—not all of them genetic. Someday, people may have the option to add genes thought to protect against high blood pressure, or certain diseases, to their genetic code.   

All CRISPR treatments are experimental at this point, and we don’t know if they’re safe. Some argue we should focus on treating those with severe diseases in the meantime. But if these new forms of CRISPR do work, they could help many others.

Read more about how CRISPR might be ready to go mainstream.

Deep Dive


Sam Altman invested $180 million into a company trying to delay death

Can anti-aging breakthroughs add 10 healthy years to the human life span? The CEO of OpenAI is paying to find out.

Forget designer babies. Here’s how CRISPR is really changing lives

The gene-editing tool is being tested in people, and the first treatment could be approved this year.

Neuroscientists listened in on people’s brains for a week. They found order and chaos.

The study shows that our brains exist between chaos and stability—a finding that could be used to help tweak them either way.

More than 200 people have been treated with experimental CRISPR therapies

But at a global genome-editing summit, exciting trial results were tempered by safety and ethical concerns.

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Illustration by Rose Wong

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